Lojuxta

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ANNEX I 
 

SUMMARY OF PRODUCT CHARACTERISTICS 
 



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This medicinal product is subject to additional monitoring. This will allow quick identification of 
new safety information. Healthcare professionals are asked to report any suspected adverse reactions. 
See section 4.8 for how to report adverse reactions. 
 
 
1. NAME OF THE MEDICINAL PRODUCT 
 
Lojuxta 5 mg hard capsules 
Lojuxta 10 mg hard capsules 
Lojuxta 20 mg hard capsules 
Lojuxta 30 mg hard capsules 
Lojuxta 40 mg hard capsules 
Lojuxta 60 mg hard capsules 
 
 
2. QUALITATIVE AND QUANTITATIVE COMPOSITION 
 
Lojuxta 5 mg hard capsules 
Each hard capsule contains lomitapide mesylate equivalent to 5 mg lomitapide. 
 
Excipient with known effect 
Each hard capsule contains 70.12 mg of lactose (as monohydrate) (see section 4.4). 
 
Lojuxta 10 mg hard capsules 
Each hard capsule contains lomitapide mesylate equivalent to 10 mg lomitapide. 
 
Excipient with known effect 
Each hard capsule contains 140.23 mg of lactose (as monohydrate) (see section 4.4). 
 
Lojuxta 20 mg hard capsules 
Each hard capsule contains lomitapide mesylate equivalent to 20 mg lomitapide. 
 
Excipient with known effect 
Each hard capsule contains 129.89 mg of lactose (as monohydrate) (see section 4.4). 
 
Lojuxta 30 mg hard capsules 
Each hard capsule contains lomitapide mesylate equivalent to 30 mg lomitapide. 
 
Excipient with known effect 
Each hard capsule contains 194.84 mg of lactose (as monohydrate) (see section 4.4). 
 
Lojuxta 40 mg hard capsules 
Each hard capsule contains lomitapide mesylate equivalent to 40 mg lomitapide. 
 
Excipient with known effect 
Each hard capsule contains 259.79 mg of lactose (as monohydrate) (see section 4.4). 
 
Lojuxta 60 mg hard capsules 
Each hard capsule contains lomitapide mesylate equivalent to 60 mg lomitapide. 
 
Excipient with known effect 
Each hard capsule contains 389.68 mg of lactose (as monohydrate) (see section 4.4). 
 
For the full list of excipients, see section 6.1. 
 
 



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3. PHARMACEUTICAL FORM 
 
 Hard, capsule. 
 
Lojuxta 5 mg hard capsules 
The capsule is an orange cap/orange body hard capsule of 19.4 mm, printed with black ink imprinted 
with “5 mg” on body and “A733” on cap. 
 
Lojuxta 10 mg hard capsules 
The capsule is an orange cap/white body hard capsule of 19.4 mm, printed with black ink imprinted 
with “10 mg” on body and “A733” on cap.  
 
Lojuxta 20 mg hard capsules 
The capsule is a white cap/white body hard capsule of 19.4 mm, printed with black ink imprinted with 
“20 mg” on body and “A733” on cap. 
 
Lojuxta 30 mg hard capsules 
The capsule is an orange cap/yellow body hard capsule of 21.6 mm, printed with black ink imprinted 
with “30 mg” on body and “A733” on cap. 
 
Lojuxta 40 mg hard capsules 
The capsule is a yellow cap/white body hard capsule of 23.4 mm, printed with black ink imprinted 
with “40 mg” on body and “A733” on cap. 
 
Lojuxta 60 mg hard capsules 
The capsule is a yellow cap/yellow body hard capsule of 23.4 mm, printed with black ink imprinted 
with “60 mg” on body and “A733” on cap. 
 
 
4. CLINICAL PARTICULARS 
 
4.1 Therapeutic indications 
 
Lojuxta is indicated as an adjunct to a low-fat diet and other lipid-lowering medicinal products with or 
without low density lipoprotein (LDL) apheresis in adult patients with homozygous familial 
hypercholesterolaemia (HoFH). 
 
Genetic confirmation of HoFH should be obtained whenever possible. Other forms of primary 
hyperlipoproteinemia and secondary causes of hypercholesterolaemia (e.g., nephrotic syndrome, 
hypothyroidism) must be excluded. 
 
4.2 Posology and method of administration 
 
Treatment with Lojuxta should be initiated and monitored by a physician experienced in the treatment 
of lipid disorders. 
 
Posology 
 
The recommended starting dose is 5 mg once daily. After 2 weeks the dose may be increased, based 
on acceptable safety and tolerability, to 10 mg and then, at a minimum of 4-week intervals, to 20 mg, 
40 mg, and to the maximum recommended dose of 60 mg (see section 4.8). 
 
The dose should be escalated gradually to minimise the incidence and severity of gastrointestinal 
adverse reactions and aminotransferase elevations. 
 



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Administration with food may increase exposure to lomitapide. It should be taken on an empty 
stomach, at least 2 hours after the evening meal because the fat content of a recent meal may adversely 
impact gastrointestinal tolerability.  
 
The occurrence and severity of gastrointestinal adverse reactions associated with the use of Lojuxta 
decreases in the presence of a low fat diet. Patients should follow a diet supplying less than 20% of 
energy from fat prior to initiating treatment, and should continue this diet during treatment. Dietary 
counselling should be provided. 
 
Patients should avoid consumption of grapefruit juice (see sections 4.4 and 4.5). 
 
For patients on a stable maintenance dose of Lojuxta who receive atorvastatin either: 

• Separate the dose of the medicinal products by 12 hours 
OR 

• Decrease the dose of Lojuxta by half. 
 
Patients on 5 mg should remain on 5 mg. 
Careful titration may then be considered according to LDL-C response and safety/tolerability. 
Upon discontinuation of atorvastatin the dose of Lojuxta should be up-titrated according to LDL-C 
response and safety/tolerability. 
 
For patients on a stable maintenance dose of Lojuxta who receive any other weak CYP3A4 inhibitor, 
separate the dose of the medicinal products (Lojuxta and the weak CYP3A4 inhibitor) by 12 hours.  
 
Consider limiting the maximum dose of Lojuxta according to desired LDL-C response.  
Exercise additional caution if administering more than 1 weak CYP3A4 inhibitor with Lojuxta. 
 
Based on observations of decreased essential fatty acid and vitamin E levels in clinical studies, 
patients should take daily dietary supplements that provide 400 IU vitamin E and approximately 
200 mg linoleic acid, 110 mg eicosapentaenoic acid (EPA), 210 mg alpha linolenic acid (ALA) and 
80 mg docosahexaenoic acid (DHA) per day, throughout treatment with Lojuxta. 
 
Special populations 
 
Elderly population 
There is limited experience with lomitapide in patients aged 65 years or older. Therefore, particular 
caution should be exercised in these patients. 
 
Since the recommended dose regimen involves starting at the low end of the dosing range and 
escalating cautiously according to individual patient tolerability, no adjustment to the dosing regimen 
is recommended for the elderly. 
 
Hepatic impairment 
Lomitapide is contraindicated in patients with moderate or severe hepatic impairment including 
patients with unexplained persistent abnormal liver function tests (see sections 4.3 and 5.2). 
 
Patients with mild hepatic impairment (Child-Pugh A) should not exceed 40 mg daily. 
 
Renal impairment  
Patients with end-stage renal disease receiving dialysis should not exceed 40 mg daily (see 
section 5.2). 
 
Paediatric population 
The safety and efficacy of lomitapide in children <18 years have not been established and the use of 
this medicinal product in children is therefore not recommended. No data are available.  
 



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Method of administration 
 
Oral use. 
 
4.3 Contraindications 
 
• Hypersensitivity to the active substance or to any of the excipients listed in section 6.1. 
• Patients with moderate or severe hepatic impairment and those with unexplained persistent 

abnormal liver function tests (see section 4.2). 
• Patients with a known significant or chronic bowel disease such as inflammatory bowel disease 

or malabsorption.  
• Concomitant administration of >40 mg simvastatin (see section 4.5). 
• Concomitant use of Lojuxta with strong or moderate cytochrome P450 (CYP) 3A4 inhibitors 

(e.g., antifungal azoles such as itraconazole, fluconazole,ketoconazole, voriconazole, 
posaconazole; macrolide antibiotics such as erythromycin or clarithromycin; ketolide antibiotics 
such as telithromycin; HIV protease inhibitors; the calcium channel blockers diltiazem and 
verapamil, and the anti-arrhythmic dronedarone [see section 4.5]). 

• Pregnancy (see section 4.6). 
 
4.4 Special warnings and precautions for use 
 
Liver enzyme abnormalities and liver monitoring 
 
Lomitapide can cause elevations in the liver enzymes alanine aminotransferase [ALT] and aspartate 
aminotransferase [AST] and hepatic steatosis (see section 5.1). There have been no concomitant or 
subsequent clinically meaningful elevations in serum bilirubin, INR, or alkaline phosphatase. The 
extent to which lomitapide-associated hepatic steatosis promotes the elevations in aminotransferase is 
unknown. The liver enzyme changes can occur at any time during therapy, but occur most often during 
dose escalation. 
Although cases of hepatic dysfunction (elevated aminotransferase with increase in bilirubin or 
International Normalized Ratio [INR]) or hepatic failure have not been reported, there is concern that 
lomitapide could induce steatohepatitis, which can progress to cirrhosis over several years. The 
clinical studies supporting the safety and efficacy of lomitapide in HoFH would have been unlikely to 
detect this adverse outcome given their size and duration. 
 
Monitoring of liver function tests  
 
Measure ALT, AST, alkaline phosphatase, total bilirubin, gamma-glutamyl transferase (gamma-GT) 
and serum albumin before initiation of treatment with Lojuxta. The medicinal product is 
contraindicated in patients with moderate or severe hepatic impairment and those with unexplained 
persistent abnormal liver function tests. If the baseline liver-related tests are abnormal, consider 
initiating the medicinal product after appropriate investigation by a hepatologist and the baseline 
abnormalities are explained or resolved.  
 
During the first year, measure liver-related tests (ALT and AST, at a minimum) prior to each increase 
in dose or monthly, whichever occurs first. After the first year, do these tests at least every 3 months 
and before any increase in dose. Decrease the dose of Lojuxta if elevations of aminotransferase are 
observed and discontinue treatment for persistent or clinically significant elevations (see Table 1 for 
specific recommendations).   
 
Dose modification based on elevated hepatic aminotransferases 
 
Table 1 summarizes recommendations for dose adjustment and monitoring for patients who develop 
elevated aminotransferase during therapy with Lojuxta.  



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Table 1: Dose adjustment and monitoring for patients with elevated aminotransferases 

ALT or AST Treatment and monitoring recommendations* 

≥3x and <5x Upper 
Limit of Normal 
(ULN) 

• Confirm elevation with a repeat measurement within one week. 

• If confirmed, reduce the dose and obtain additional liver-related tests if 
not already measured (such as alkaline phosphatase, total bilirubin, and 
INR).   

• Repeat tests weekly and withhold dosing if there are signs of abnormal 
liver function (increase in bilirubin or INR), if aminotransferase levels 
rise above 5x ULN, or if aminotransferase levels do not fall below 3x 
ULN within approximately 4 weeks. Refer patients with persistent 
elevations in aminotransferase >3x ULN to a hepatologist for further 
investigation. 

• If resuming Lojuxta after aminotransferase levels resolve to <3x ULN, 
consider reducing the dose and monitor liver-related tests more 
frequently.  

≥5x ULN • Withhold dosing and obtain additional liver-related tests if not already 
measured (such as alkaline phosphatase, total bilirubin, and INR).  If 
aminotransferase levels do not fall below 3x ULN within approximately 
4 weeks refer the patient to a hepatologist for further investigation. 

• If resuming Lojuxta after aminotransferase levels resolve to <3x ULN, 
reduce the dose and monitor liver-related tests more frequently. 

*Recommendations based on an ULN of approximately 30-40 international units/L. 
 
If aminotransferase elevations are accompanied by clinical symptoms of liver injury (such as nausea, 
vomiting, abdominal pain, fever, jaundice, lethargy, flu-like symptoms), increases in bilirubin ≥2x 
ULN, or active liver disease, discontinue treatment with Lojuxta and refer the patient to a hepatologist 
for further investigation. 
 
Reintroduction of treatment may be considered if the benefits are considered to outweigh the risks 
associated with potential liver disease.  
 
Hepatic steatosis and risk of progressive liver disease 
 
Consistent with the mechanism of action of lomitapide, most treated patients exhibited increases in 
hepatic fat content. In an open-label Phase 3 study, 18 of 23 patients with HoFH developed hepatic 
steatosis (hepatic fat >5.56%) as measured by nuclear magnetic resonance spectroscopy (MRS) (see 
section 5.1). The median absolute increase in hepatic fat was 6% after both 26 weeks and 78 weeks of 
treatment, from 1% at baseline, measured by MRS. Hepatic steatosis is a risk factor for progressive 
liver disease including steatohepatitis and cirrhosis. The long term consequences of hepatic steatosis 
associated with lomitapide treatment are unknown. Clinical data suggest that hepatic fat accumulation 
is reversible after stopping treatment with Lojuxta, but whether histological sequelae remain is 
unknown, especially after long-term use.  
 
Monitoring for evidence of progressive liver disease. 
 
Regular screening for steatohepatitis/fibrosis should be performed at baseline and on an annual basis 
using the following imaging and biomarker evaluations: 
 

• Imaging for tissue elasticity, e.g. Fibroscan, acoustic radiation force impulse (ARFI), or 
magnetic resonance (MR) elastography 

 
• Gamma-GT and serum albumin to detect possible liver injury 
 



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• At least one marker from each of the following categories:  
 

• High sensitivity C-reactive protein (hs-CRP), erythrocyte sedimentation rate (ESR), CK-
18 Fragment, NashTest (liver inflammation) 

• Enhanced Liver Fibrosis (ELF) panel, Fibrometer, AST/ALT ratio, Fib-4 score, Fibrotest 
(liver fibrosis) 

  
The performance of these tests and their interpretation should involve collaboration between the 
treating physician and the hepatologist. Patients with results suggesting the presence of steatohepatitis 
or fibrosis should be considered for liver biopsy.  
 
If a patient has biopsy-proven steatohepatitis or fibrosis, the benefit-risk should be reassessed and 
treatment stopped if necessary. 
 
Dehydration 
 
Post-marketing reports of dehydration and hospitalisation in patients treated with lomitapide have been 
reported. Patients treated with lomitapide should be advised of the potential risk of dehydration in 
relation to gastrointestinal adverse reactions and take precautions to avoid fluid depletion. 
 
Concomitant use of CYP3A4 inhibitors 
 
Lomitapide appears to be a sensitive substrate for CYP3A4 metabolism. CYP3A4 inhibitors increase 
the exposure of lomitapide, with strong inhibitors increasing exposure approximately 27-fold. 
Concomitant use of moderate or strong CYP3A4 inhibitors with Lojuxta is contraindicated (see 
section 4.3). In the lomitapide clinical studies, one patient with HoFH developed markedly elevated 
aminotransferase (ALT 24x ULN, AST 13x ULN) within days of initiating the strong CYP3A4 
inhibitor clarithromycin. If treatment with moderate or strong CYP3A4 inhibitors is unavoidable, 
Lojuxta should be stopped during the course of treatment. 
 
Weak CYP3A4 inhibitors are expected to increase the exposure of lomitapide when taken 
simultaneously. When administered with atorvastatin, the dose of Lojuxta should either be taken 
12 hours apart or be decreased by half (see section 4.2). The dose of Lojuxta should be administered 
12 hours apart from any other weak CYP3A4 inhibitor. 
 
Concomitant use of CYP3A4 inducers 
 
Medicinal products that induce CYP3A4 would be expected to increase the rate and extent of 
metabolism of lomitapide. CYP3A4 inducers exert their effect in a time-dependent manner, and may 
take at least 2 weeks to reach maximal effect after introduction. Conversely, on discontinuation, 
CYP3A4 induction may take at least 2 weeks to decline. 
 
Co-administration of a CYP3A4 inducer is expected to reduce the effect of lomitapide. Any impact on 
efficacy is likely to be variable. When co-administering CYP3A4 inducers (i.e. aminoglutethimide, 
nafcillin, non-nucleoside reverse transcriptase inhibitors, phenobarbital, rifampicin, carbamazepine, 
pioglitazone, glucocorticoids, modafinil and phenytoin) with Lojuxta, the possibility of a drug-drug 
interaction affecting efficacy should be considered. The use of St. John’s Wort should be avoided with 
Lojuxta. 
 
It is recommended to increase the frequency of LDL-C assessment during such concomitant use and 
consider increasing the dose of Lojuxta to ensure maintenance of the desired level of efficacy if the 
CYP3A4 inducer is intended for chronic use. On withdrawal of a CYP3A4 inducer, the possibility of 
increased exposure should be considered and a reduction in the dose of Lojuxta may be necessary. 
 



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Concomitant use of HMG-CoA reductase inhibitors (‘statins’) 
 
Lomitapide increases plasma concentrations of statins. Patients receiving Lojuxta as adjunctive 
therapy to a statin should be monitored for adverse events that are associated with the use of high 
doses of statins. Statins occasionally cause myopathy. In rare cases, myopathy may take the form of 
rhabdomyolysis with or without acute renal failure secondary to myoglobinuria, and can lead to 
fatality. All patients receiving lomitapide in addition to a statin should be advised of the potential 
increased risk of myopathy and told to report promptly any unexplained muscle pain, tenderness, or 
weakness. Doses of simvastatin >40 mg should not be used with Lojuxta (see section 4.3). 
 
Grapefruit juice 
 
Grapefruit juice must be omitted from the diet while patients are treated with Lojuxta. 
 
Risk of supratherapeutic or subtherapeutic anticoagulation with coumarin based anticoagulants 
 
Lomitapide increases the plasma concentrations of warfarin. Increases in the dose of Lojuxta may lead 
to supratherapeutic anticoagulation, and decreases in the dose may lead to subtherapeutic 
anticoagulation. Difficulty controlling INR contributed to early discontinuation from the Phase 3 study 
for one of five patients taking concomitant warfarin. Patients taking warfarin should undergo regular 
monitoring of the INR, especially after any changes in the dose of Lojuxta. The dose of warfarin 
should be adjusted as clinically indicated. 
 
Use of alcohol 
 
Alcohol may increase levels of hepatic fat and induce or exacerbate liver injury. In the Phase 3 study, 
3 of 4 patients with ALT elevations >5x ULN reported alcohol consumption beyond the limits 
recommended in the protocol. The use of alcohol during lomitapide treatment is not recommended. 
 
Hepatotoxic agents 
 
Caution should be exercised when Lojuxta is used with other medicinal products known to have 
potential for hepatotoxicity, such as isotretinoin, amiodarone, acetaminophen (>4 g/day for 
≥3 days/week), methotrexate, tetracyclines, and tamoxifen. The effect of concomitant administration 
of lomitapide with other hepatotoxic medicine is unknown. More frequent monitoring of liver-related 
tests may be warranted. 
 
Reduced absorption of fat-soluble vitamins and serum fatty acids 
 
Given its mechanism of action in the small intestine, lomitapide may reduce the absorption of 
fat-soluble nutrients. In the Phase 3 study, patients were provided daily dietary supplements of vitamin 
E, linoleic acid, ALA, EPA and DHA. In this study, the median levels of serum vitamin E, ALA, 
linoleic acid, EPA, DHA, and arachidonic acid decreased from baseline to Week 26 but remained 
above the lower limit of the reference range. Adverse clinical consequences of these reductions were 
not observed with lomitapide treatment of up to 78 weeks. Patients treated with Lojuxta should take 
daily supplements that contain 400 international units vitamin E and approximately 200 mg linoleic 
acid, 210 mg ALA, 110 mg EPA, and 80 mg DHA. 
 
Contraception measures in women of child-bearing potential 
 
Before initiating treatment in women of child-bearing potential, appropriate advice on effective 
methods of contraception should be provided, and effective contraception initiated. Patients taking 
oestrogen-based oral contraceptives should be advised about possible loss of effectiveness due to 
diarrhoea and/or vomiting (see section 4.5). Oestrogen-containing oral contraceptives are weak 
CYP3A4 inhibitors (see section 4.2).  
 



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Patients should be advised to immediately contact their physician and stop taking Lojuxta if they 
become pregnant (see section 4.6). 
 
Lactose 
 
Lojuxta contains lactose. Patients with rare hereditary problems of galactose intolerance, total-lactase 
deficiency or glucose-galactose malabsorption should not take this medicinal product. 
 
4.5 Interaction with other medicinal products and other forms of interaction 
 
Effects of other medicinal products on lomitapide and other forms of interaction 

Table 2: Interactions between Lojuxta and other medicinal products and other forms of 
interaction 

Medicinal products Effects on lomitapide levels Recommendation concerning 
co-administration with Lojuxta 

Inhibitors of CYP3A4 

 

When lomitapide 60 mg was 
co-administered with 
ketoconazole 200 mg twice 
daily, a strong inhibitor of 
CYP3A4, lomitapide AUC 
increased approximately 27-fold 
and Cmax increased 
approximately 15-fold. 

Interactions between moderate 
CYP3A4 inhibitors and 
lomitapide have not been 
studied.  

Moderate CYP3A4 inhibitors are 
predicted to have a substantial 
impact on lomitapide’s 
pharmacokinetics. Concomitant 
use of moderate CYP3A4 
inhibitors are expected to 
increase lomitapide exposure by 
4-10 fold based on the results of 
the study with the strong 
CYP3A4 inhibitor ketoconazole 
and on historical data for the 
model CYP3A4 probe 
midazolam. 

Weak CYP3A4 inhibitors are 
expected to increase the 
exposure of lomitapide when 
taken simultaneously. 

When lomitapide 20 mg was 
co-administered simultaneously 
with atorvastatin, a weak 
CYP3A4 inhibitor, lomitapide 
AUC and Cmax increased 
approximately 2-fold. When the 
dose of lomitapide was taken 
12 hours apart from atorvastatin, 

Use of strong or moderate inhibitors of 
CYP3A4 is contraindicated with 
Lojuxta. If treatment with antifungal 
azoles (e.g., itraconazole, ketoconazole, 
fluconazole, voriconazole, 
posaconazole); the antiarrhythmic 
dronedarone; macrolide antibiotics (e.g., 
erythromycin, clarithromycin); ketolide 
antibiotics (e.g., telithromycin); HIV 
protease inhibitors; the calcium channel 
blockers diltiazem and verapamil is 
unavoidable, therapy with Lojuxta 
should be suspended during the course of 
treatment (see sections 4.3 and 4.4). 

Grapefruit juice is a moderate inhibitor 
of CYP3A4 and is expected to 
substantially increase exposure to 
lomitapide. Patients taking Lojuxta 
should avoid consumption of grapefruit 
juice. 

When administered with atorvastatin, the 
dose of Lojuxta should either be taken 
12 hours apart or be decreased by half 
(see section 4.2). The dose of Lojuxta 
should be taken 12 hours apart from any 
other concomitant weak CYP3A4 
inhibitors. Examples of weak CYP3A4 
inhibitors include: alprazolam, 
amiodarone, amlodipine, atorvastatin, 
azithromycin, bicalutamide, cilostazol, 
cimetidine, ciclosporin, clotrimazole, 
fluoxetine, fluvoxamine, fosaprepitant, 
ginkgo, goldenseal, isoniazid, ivacaftor, 
lacidipine, lapatinib, linagliptin, 
nilotinib, oestrogen-containing oral 
contraceptives, pazopanib, peppermint 
oil, propiverine, ranitidine, ranolazine, 
roxithromycin, Seville oranges, 
tacrolimus, ticagrelor and tolvaptan. This 



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Medicinal products Effects on lomitapide levels Recommendation concerning 
co-administration with Lojuxta 

no clinically meaningful increase 
in lomitapide exposure was 
observed. 

When lomitapide 20 mg was co-
administered simultaneously or 
12 hours apart with ethinyl 
estradiol/norgestimate, a weak 
CYP3A4 inhibitor, no clinically 
meaningful increase in 
lomitapide exposure was 
observed. 

list is not intended to be comprehensive 
and prescribers should check the 
prescribing information of medicinal 
products to be co-administered with 
Lojuxta for potential CYP3A4 mediated 
interactions.  

The effect of administration of more than 
one weak CYP3A4 inhibitor has not 
been tested, but the effect on the 
exposure of lomitapide is expected to be 
greater than for co-administration of the 
individual inhibitors with lomitapide. 

Exercise additional caution if 
administering more than 1 weak 
CYP3A4 inhibitor with Lojuxta. 

Inducers of CYP3A4 Medicines that induce CYP3A4 
would be expected to increase 
the rate and extent of 
metabolism of lomitapide. 
Consequently, this would reduce 
the effect of lomitapide. Any 
impact on efficacy is likely to be 
variable. 

When co-administering CYP3A4 
inducers (i.e., aminoglutethimide, 
nafcillin, non-nucleoside reverse 
transcriptase inhibitors, phenobarbital, 
rifampicin, carbamazepine, pioglitazone, 
St John’s Wort, glucocorticoids, 
modafinil and phenytoin) with Lojuxta, 
the possibility of a drug-drug interaction 
affecting efficacy should be considered. 
It is recommended to increase the 
frequency of LDL-C assessment during 
such concomitant use and consider 
increasing the dose of Lojuxta to ensure 
maintenance of the desired level of 
efficacy if the CYP3A4 inducer is 
intended for chronic use. 

Bile acid sequestrants Lomitapide has not been tested 
for interaction with bile acid 
sequestrants (resins such as 
colesevelam and 
cholestyramine).  

Because bile acid sequestrants can 
interfere with the absorption of oral 
medicines, bile acid sequestrants should 
be taken at least 4 hours before or at least 
4 hours after Lojuxta. 

 
Effects of lomitapide on other medicinal products 
 
HMG-CoA Reductase Inhibitors (“Statins”): Lomitapide increases plasma concentrations of statins. 
When lomitapide 60 mg was administered to steady state prior to simvastatin 40 mg, simvastatin acid 
AUC and Cmax increased 68% and 57%, respectively. When lomitapide 60 mg was administered to 
steady state prior to atorvastatin 20 mg, atorvastatin acid AUC and Cmax increased 52% and 63%, 
respectively. When lomitapide 60 mg was administered to steady state prior to rosuvastatin 20 mg, 
rosuvastatin Tmax increased from 1 to 4 hours, AUC was increased 32%, and its Cmax was unchanged.  
The risk of myopathy with simvastatin is dose related. Use of Lojuxta is contraindicated in patients 
treated with high doses of simvastatin (>40 mg) (see sections 4.3 and 4.4). 
 
Coumarin anticoagulants: When lomitapide 60 mg was administered to steady state and 6 days 
following warfarin 10 mg, INR increased 1.26-fold. AUCs for R(+)-warfarin and S(-)-warfarin 
increased 25% and 30%, respectively. Cmax for R(+)-warfarin and S(-)-warfarin increased 14% and 



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15%, respectively. In patients taking coumarins (such as warfarin) and Lojuxta concomitantly, INR 
should be determined before starting Lojuxta and monitored regularly with dosage of coumarins 
adjusted as clinically indicated (see section 4.4). 
 
Fenofibrate, niacin and ezetimibe: When lomitapide was administered to steady state prior to 
micronised fenofibrate 145 mg, extended release niacin 1000 mg, or ezetimibe 10 mg, no clinically 
significant effects on the exposure of any of these medicinal products were observed. No dose 
adjustments are required when co-administered with Lojuxta. 
 
Oral contraceptives: When lomitapide 50 mg was administered to steady state along with an 
oestrogen-based oral contraceptive, no clinically meaningful or statistically significant impact on the 
pharmacokinetics of the components of the oral contraceptive (ethinyl estradiol and 17-deacetyl 
norgestimate, the metabolite of norgestimate) was observed. Lomitapide is not expected to directly 
influence the efficacy of oestrogen based oral contraceptives; however diarrhoea and/or vomiting may 
reduce hormone absorption. In cases of protracted or severe diarrhoea and/or vomiting lasting more 
than 2 days, additional contraceptive measures should be used for 7 days after resolution of symptoms. 
 
P-gp substrates: Lomitapide inhibits P-gp in vitro, and may increase the absorption of P-gp substrates. 
Coadministration of Lojuxta with P gp substrates (such as aliskiren, ambrisentan, colchicine, 
dabigatran etexilate, digoxin, everolimus, fexofenadine, imatinib, lapatinib, maraviroc, nilotinib, 
posaconazole, ranolazine, saxagliptin, sirolimus, sitagliptin, talinolol, tolvaptan, topotecan) may 
increase the absorption of P gp substrates. Dose reduction of the P gp substrate should be considered 
when used concomitantly with Lojuxta. 
 
In vitro assessment of drug interactions: Lomitapide inhibits CYP3A4. Lomitapide does not induce 
CYPs 1A2, 3A4, or 2B6, and does not inhibit CYPs 1A2, 2B6, 2C9, 2C19, 2D6, or 2E1. Lomitapide is 
not a P-gp substrate but does inhibit P-gp. Lomitapide does not inhibit breast cancer resistance protein 
(BCRP). 
 
4.6 Fertility, pregnancy and lactation 
 
Use in women of child-bearing potential 
 
Before initiating treatment in women of child-bearing potential, the absence of pregnancy should be 
confirmed, appropriate advice on effective methods of contraception provided, and effective 
contraception initiated. Patients taking oestrogen-based oral contraceptives should be advised about 
possible loss of effectiveness due to diarrhoea and/or vomiting. Additional contraceptive measures 
should be used until resolution of symptoms (see section 4.5). 
 
Pregnancy 
 
Lojuxta is contraindicated during pregnancy. There are no reliable data on its use in pregnant women. 
Animal studies have shown developmental toxicity (teratogenicity, embryotoxicity, see section 5.3). 
The potential risk for humans is unknown.  
 
Breast-feeding 
 
It is not known whether lomitapide is excreted into human milk. Because of the potential for adverse 
effects based on findings in animal studies with lomitapide (see section 5.3), a decision should be 
made whether to discontinue breast-feeding or discontinue the medicinal product, taking into account 
the importance of the medicinal product to the mother. 
 



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Fertility  
 
No adverse effects on fertility were observed in male and female rats administered lomitapide at 
systemic exposures (AUC) estimated to be 4 to 5 times higher than in humans at the maximum 
recommended human dose (see section 5.3).  
 
4.7 Effects on ability to drive and use machines 
 
Lojuxta has minor influence on the ability to drive and use machines.  
 
4.8 Undesirable effects 
 
Summary of the safety profile 
 
The most serious adverse reactions during treatment were liver aminotransferase abnormalities (see 
section 4.4). 
 
The most common adverse reactions were gastrointestinal effects. Gastrointestinal adverse reactions 
were reported by 27 (93%) of 29 patients in the Phase 3 clinical study. Diarrhoea occurred in 79% of 
patients, nausea in 65%, dyspepsia in 38%, and vomiting in 34%. Other reactions reported by at least 
20% of patients include abdominal pain, abdominal discomfort, abdominal distension, constipation, 
and flatulence. Gastrointestinal adverse reactions occurred more frequently during the dose escalation 
phase of the study and decreased once patients established the maximum tolerated dose of lomitapide. 
 
Gastrointestinal adverse reactions of severe intensity were reported by 6 (21%) of 29 patients in the 
Phase 3 clinical study, with the most common being diarrhoea (4 patients, 14%); vomiting (3 patients, 
10%); and abdominal pain, distension, and/or discomfort (2 patients, 7%). Gastrointestinal reactions 
contributed to the reasons for early discontinuation from the study for 4 (14%) patients. 
 
The most commonly reported adverse reactions of severe intensity were diarrhoea (4 subjects, 14%), 
vomiting (3 patients, 10%), and abdominal distension and ALT increased (2 subjects each, 7%). 
 
Tabulated list of adverse reactions 
 
Frequency of the adverse reactions is defined as: very common (≥1/10), common (≥1/100 to <1/10), 
uncommon (≥1/1,000 to <1/100), rare (≥1/10,000 to <1/1,000), very rare (<1/10,000), not known 
(cannot be estimated from the available data). 
 
Table 3 lists all adverse reactions reported across the 35 patients treated in the Phase 2 Study UP1001 
and in the Phase 3 Study UP1002/AEGR-733-005 or its extension study AEGR-733-012.  



13 

Table 3: Frequency of adverse reactions in HoFH patients 
System Organ Class Frequency Adverse reaction 
Infections and infestations Common Gastroenteritis 
Metabolism and nutrition 
disorders  

Very common Decreased appetite 
Not known Dehydration 

Nervous system disorders Common Dizziness 
Headache 
Migraine 

Gastrointestinal disorders Very common Diarrhoea 
Nausea 
Vomiting 
Abdominal discomfort 
Dyspepsia 
Abdominal pain 
Abdominal pain upper 
Flatulence  
Abdominal distension 
Constipation 

Common Gastritis  
Rectal tenesmus 
Aerophagia 
Defaecation urgency 
Eructation 
Frequent bowel movements 
Gastric dilatation 
Gastric disorder 
Gastrooesophageal reflux disease 
Haemorrhoidal haemorrhage 
Regurgitation 

Hepatobiliary disorders Common Hepatic steatosis  
Hepatotoxicity 
Hepatomegaly 

Skin and subcutaneous tissue 
disorders 

Common Ecchymosis 
Papule 
Rash erythematous 
Xanthoma 

Not known Alopecia 
Musculoskeletal and connective 
tissue disorders 

Not known Myalgia 

General disorders and 
administration site conditions 

Common Fatigue 

Investigations Very common Alanine aminotransferase increased 
Aspartate aminotransferase increased 
Weight decreased 

Common International normalised ratio 
increased 
Blood alkaline phosphatase increased 
Blood potassium decreased 
Carotene decreased 
International normalised ratio 
abnormal 
Liver function test abnormal 
Prothrombin time prolonged 
Transaminases increased 
Vitamin E decreased 
Vitamin K decreased 



14 

 
Table 4 lists all adverse reactions for subjects who received lomitapide monotherapy (N=291) treated 
in Phase 2 studies in subjects with elevated LDL-C (N=462). 

Table 4: Frequency of adverse reactions in elevated LDL-C patients 
System Organ Class Frequency Adverse reaction 
Infections and infestations Uncommon Gastroenteritis 

Gastrointestinal infection 
Influenza 
Nasopharyngitis 
Sinusitis 

Blood and lymphatic system 
disorders 

Uncommon Anaemia 

Metabolism and nutrition 
disorders  

Common Decreased appetite  
Uncommon Dehydration 

Increased appetite 
Nervous system disorders Uncommon Paraesthesia 

Somnolence 
Eye disorders Uncommon Eye swelling 
Ear and labyrinth disorders Uncommon Vertigo 
Respiratory, thoracic and 
mediastinal disorders 

Uncommon Pharyngeal lesion 
Upper-airway cough syndrome 

Gastrointestinal disorders Very common Diarrhoea 
Nausea  
Flatulence  

Common Abdominal pain upper 
Abdominal distension 
Abdominal pain 
Vomiting 
Abdominal discomfort 
Dyspepsia 
Eructation 
Abdominal pain lower 
Frequent bowel movements 

 

Uncommon Dry mouth 
Faeces hard 
Gastrooeosophageal reflux disease 
Abdominal tenderness 
Epigastric discomfort 
Gastric dilatation 
Haematemesis 
Lower gastrointestinal haemorrhage 
Reflux oesophagitis 

Hepatobiliary disorders Uncommon Hepatomegaly 
Skin and subcutaneous tissue 
disorders 

Uncommon Blister 
Dry skin 
Hyperhidrosis 

Musculoskeletal and connective 
tissue disorders 

Common Muscle spasms 
Uncommon Arthralgia 

Myalgia 
Pain in extremity 
Joint swelling 
Muscle twitching 

Renal and urinary disorders Uncommon Haematuria 



15 

System Organ Class Frequency Adverse reaction 
General disorders and 
administrative site conditions 

Common Fatigue 
Asthenia 

Uncommon Chest pain 
Chills 
Early satiety 
Gait disturbance 
Malaise 
Pyrexia 

Investigations Common Alanine aminotransferase increased 
Aspartate aminotransferase increased 
Hepatic enzyme increased 
Liver function test abnormal 
Neutrophil count decreased 
White blood cell count decreased 

Uncommon Weight decreased 
Blood bilirubin increased 
Gamma-glutamyltransferase increased 
Neutrophil percentage increased 
Protein urine 
Prothrombin time prolonged 
Pulmonary function test abnormal 
White blood cell count increased 

 
Reporting of suspected adverse reactions 
Reporting suspected adverse reactions after authorisation of the medicinal product is important. It 
allows continued monitoring of the benefit/risk balance of the medicinal product. Healthcare 
professionals are asked to report any suspected adverse reactions via the national reporting system 
listed in Appendix V. 
 
4.9 Overdose 
 
There is no specific treatment in the event of overdose. In the event of overdose, the patient should be 
treated symptomatically and supportive measures instituted as required. Liver related tests should be 
monitored. Haemodialysis is unlikely to be beneficial given that lomitapide is highly protein bound. 
 
In rodents, single oral doses of lomitapide ≥600 times higher than the maximum recommended human 
dose (1 mg/kg) were well tolerated. The maximum dose administered to human subjects in clinical 
studies was 200 mg as a single dose; there were no adverse reactions. 
 
 
5. PHARMACOLOGICAL PROPERTIES 
 
5.1  Pharmacodynamic properties 
 
Pharmacotherapeutic group: Lipid modifying agents, other lipid modifying agents, ATC code: 
C10AX12 

 
Mechanism of action 
 
Lomitapide is a selective inhibitor of microsomal transfer protein (MTP), an intracellular lipid-transfer 
protein that is found in the lumen of the endoplasmic reticulum and is responsible for binding and 
shuttling individual lipid molecules between membranes. MTP plays a key role in the assembly of 
apo B containing lipoproteins in the liver and intestines. Inhibition of MTP reduces lipoprotein 
secretion and circulating concentrations of lipoprotein-borne lipids including cholesterol and 
triglycerides. 
 

http://www.ema.europa.eu/docs/en_GB/document_library/Template_or_form/2013/03/WC500139752.doc


16 

Clinical efficacy and safety 
 
A single arm, open-label study (UP1002/AEGR-733-005) evaluated the efficacy and safety of 
lomitapide when co-administered with a low-fat diet and other lipid-lowering therapies in adult 
patients with HoFH. Patients were instructed to maintain a low-fat diet (<20% calories from fat) and 
their lipid-lowering therapies at study entry, including apheresis if applicable, from 6 weeks prior to 
baseline through at least Week 26. The dose of lomitapide was escalated from 5 mg to an individually 
determined maximum tolerated dose up to 60 mg. After Week 26, patients remained on lomitapide to 
determine the effects of longer-term treatment and were allowed to change background lipid-lowering 
therapies. The study provided for a total of 78 weeks of treatment. 
 
Twenty-nine patients were enrolled, of whom 23 completed through Week 78. Sixteen males (55%) 
and 13 females (45%) were included with a mean age of 30.7 years, ranging from 18 to 55 years. The 
mean dose of lomitapide was 45 mg at Week 26 and 40 mg at Week 78. At Week 26, the mean percent 
change in LDL-C from baseline of LDL-C was -40% (p<0.001) in the Intent to Treat (ITT) population. 
Mean percent change from baseline through Week 26 using last observation carried forward (LOCF) 
to each assessment is shown in Figure 1. 
 

Figure 1: Mean percent changes from baseline in LDL-C in the major effectiveness study 
UP1002/AEGR-733-005 through Week 26 (the Primary Endpoint) using LOCF 
to each assessment (N=29) 

-8

-17

-25

-38

-43

-39 -40

-50

-45

-40

-35

-30

-25

-20

-15

-10

-5

0

Week 0 Week 2 Week 6 Week 10 Week 14 Week 18 Week 22 Week 26

M
ea

n 
Pe

rc
en

t C
ha

ng
e f

ro
m

 B
as

el
in

e i
n 

LD
L-

C

Study Week  
 
Changes in lipids and lipoproteins through Week 26 and Week 78 of lomitapide treatment are 
presented in Table 5. 



17 

Table 5: Absolute values and percent changes from baseline to Weeks 26 and 78 in lipids 
and lipoproteins (major effectiveness study UP1002/AEGR-733-005) 

Parameter (units) Baseline Week 26/LOCF (N=29) Week 78 (N=23) 

Mean 
(SD) 

Mean 
(SD) 

% 
Change p-valueb 

Mean 
(SD) 

% 
Change p-valueb 

LDL-C, direct  
(mg/dL)  

336  
(114) 

190  
(104) -40 <0.001 

210  
(132) -38 <0.001 

Total Cholesterol (TC) 
(mg/dL)  

430  
(135) 

258  
(118) -36 <0.001 

281  
(149) -35 <0.001 

Apolipoprotein B 
(apo B) (mg/dL) 

259  
(80) 

148  
(74) -39 <0.001 

151  
(89) -43 <0.001 

Triglycerides (TG) 
(mg/dL)a 92 57 -45 0.009 59 -42 0.012 

Non high-density 
lipoprotein cholesterol 
(Non-HDL-C) (mg/dL)  

386  
(132) 

217  
(113) -40 <0.001 

239 
(146) -39 <0.001 

Very-low-density 
lipoprotein cholesterol 
(VLDL-C) (mg/dL)  

21  
(10) 

13  
(9) -29 0.012 

16  
(15) -31 0.013 

Lipoprotein (a) (Lp(a)) 
(nmol/L)a 66 61 -13 0.094 72 -4 <0.842 

High-density lipoprotein 
cholesterol (HDL-C) 
(mg/dL)  

44  
(11) 

41  
(13) -7 0.072 

43  
(12) -4.6 0.246 

a Median presented for TG and Lp(a). p-value is based on the mean percent change 
b p-value on the mean percent change from baseline based on paired t-test 

At both Week 26 and Week 78, there were significant reductions in LDL-C, TC, apo B, TG, 
non-HDL-C, VLDL-C and changes in HDL-C trended lower at Week 26 and returned to baseline 
levels by Week 78.  
 
The effect of Lojuxta on cardiovascular morbidity and mortality has not been determined. 
 
At baseline, 93% were on a statin, 76% were on ezetimibe, 10% on niacin, 3% on a bile acid 
sequestrant and 62% were receiving apheresis. Fifteen of 23 (65%) patients had their lipid-lowering 
treatment reduced by Week 78, including planned and unplanned reductions/interruptions. Apheresis 
was discontinued in 3 out of 13 patients who were on it at Week 26, and frequency was reduced in 
3 patients while maintaining low LDL-C levels through Week 78. The clinical benefit of reductions in 
background lipid-lowering therapy, including apheresis, is not certain. 
 
Of the 23 patients who completed through Week 26, 19 (83%) had LDL-C reductions ≥25% with 
8 (35%) having LDL-C <100 mg/dL and 1 having LDL-C <70 mg/dL at that time point. 
 
In this study, 10 patients experienced elevations in AST and/or ALT >3 x ULN (see Table 6).  



18 

Table 6: Highest liver function test results post first dose (major effectiveness study 
UP1002/AEGR-733-005) 

Parameter/Abnormality N (%) 

ALT  

Number of Patients with Assessments 29 

>3 to ≤5 x ULN 6 (20.7) 

>5 to ≤10 x ULN 3 (10.3) 

>10 to ≤20 x ULN 1 (3.4) 

>20 x ULN 0 

AST  

Number of Patients with Assessments 29 

>3 to ≤5 x ULN 5 (17.2) 

>5 to ≤10 x ULN 1 (3.4) 

>10 to ≤20 x ULN 0 

>20 x ULN 0 
 
Elevations in ALT and/or AST >5 x ULN were managed with a dose reduction or temporary 
suspension of lomitapide dosing, and all patients were able to continue with study drug treatment. No 
clinically meaningful elevations in total bilirubin or alkaline phosphatase were observed. Hepatic fat 
was prospectively measured using MRS in all eligible patients during the clinical study (Table 7). Data 
from individuals who had repeat measurements after stopping lomitapide show that hepatic fat 
accumulation is reversible, but whether histological sequelae remain is unknown. 

Table 7: Maximum categorical changes in % hepatic fat (major effectiveness study 
UP1002/AEGR-733-005) 

Maximum absolute increase 
in % hepatic fat 

Efficacy phase 
weeks 0-26 
N (%) 

Safety phase 
weeks 26-78 
N (%) 

Entire trial  
weeks 0-78 
N (%) 

Number of evaluable patients 22 22 23 

≤5% 9 (41) 6 (27) 5 (22) 

>5% to ≤10% 6 (27) 8 (36) 8 (35) 

>10% to ≤15% 4 (18) 3 (14) 4 (17) 

>15% to ≤20% 1 (5) 4 (18) 3 (13) 

>20% to ≤25% 1 (5) 0 1 (4) 

>25% 1 (5) 1 (5) 2 (9) 

 
The European Medicines Agency has deferred the obligation to submit the results of studies with 
Lojuxta in one or more subsets of the paediatric population in HoFH (see section 4.2 for information 
on paediatric use). 
 
This medicinal product has been authorised under ‘exceptional circumstances’. This means that due to 
the rarity of the disease it has not been possible to obtain complete information on this medicinal 
product. 
The European Medicines Agency will review any new information which may become available every 
year and this SmPC will be updated as necessary. 



19 

5.2 Pharmacokinetic properties 
 
Absorption 
 
The absolute oral bioavailability of lomitapide is 7%. Absorption is not limited by penetration of the 
active substance across the intestinal barrier but is predominantly influenced by an extensive first pass 
effect. Peak plasma concentrations of lomitapide were reached 4-8 hours following oral dosing. 
Lomitapide pharmacokinetics is approximately dose-proportional for oral single doses in the 
therapeutic range. Doses higher than 60 mg suggest a trend toward nonlinearity and are not 
recommended.  
 
Upon multiple dosing Cmax and AUC increased in approximate proportion to lomitapide dose. Cmax and 
AUC were increased following either a high-fat meal (77% and 58%, respectively) or low fat meal 
(70% and 28%, respectively). Accumulation of lomitapide in plasma was consistent with that 
predicted after a single dose following once daily oral dosing above 25 mg for up to 4 weeks. 
Inter-individual variability in lomitapide AUC was approximately 50%. 
 
At steady state the accumulation of lomitapide was 2.7 at 25 mg and 3.9 at 50 mg. 
 
Distribution 
 
Following intravenous administration, the volume of distribution of lomitapide was high 
(mean=1200 litres) despite a high degree (>99.8%) of binding to plasma protein.   In animal studies 
lomitapide was highly concentrated (200-fold) in the liver. 
 
Biotransformation 
 
Lomitapide is extensively metabolised, predominantly by CYP3A4. CYP isoforms 2E1, 1A2, 2B6, 
2C8, and 2C19 are involved to a lesser extent and isoforms 2D6 and 2C9 are not involved in the 
metabolism of lomitapide.  
 
Elimination 
 
Following administration of a radiolabeled oral solution dose to healthy subjects, 93% of the 
administered dose was recovered in urine and faeces. Approximately 33% of the radioactivity was 
excreted in urine as metabolites. The remainder was excreted in faeces, primarily as oxidised 
metabolites. The elimination half-life of lomitapide was approximately 29 hours. 
 
Special populations 
 
Data in the pivotal clinical study were analysed with respect to the impact of potential covariates on 
lomitapide exposure. Of the parameters examined (race, body mass index (BMI), gender, weight, age), 
only BMI could be classified as a potential covariate.  
 
Age and gender 
 
There was no clinically relevant effect of age (18-64 years) or gender on the pharmacokinetics of 
lomitapide. 
 
Race 
 
No dose adjustment is required for Caucasian or Latino patients. There is insufficient information to 
determine if Lojuxta requires dose adjustment in other races. However, since the medicinal product is 
dosed in an escalating fashion according to individual patient safety and tolerability, no adjustment to 
the dosing regimen is recommended based on race. 
 



20 

Renal insufficiency 
 
In the renal impairment population, lomitapide was only studied in patients with end-stage renal 
disease (ESRD). A pharmacokinetic study in patients with ESRD undergoing hemodialysis 
demonstrated a 36% increase in mean lomitapide plasma concentration compared to matched healthy 
controls. The terminal half-life of lomitapide was not affected. 
 
Hepatic insufficiency 
 
A single-dose, open-label study was conducted to evaluate the pharmacokinetics of 60 mg lomitapide 
in healthy volunteers with normal hepatic function compared with patients with mild (Child-Pugh A) 
and moderate (Child-Pugh B) hepatic impairment. In patients with moderate hepatic impairment, 
lomitapide AUC and Cmax were 164% and 361% higher, respectively, compared with healthy 
volunteers. In patients with mild hepatic impairment, lomitapide AUC and Cmax were 47% and 4% 
higher, respectively, compared with healthy volunteers. Lojuxta has not been studied in patients with 
severe hepatic impairment (Child-Pugh score 10-15). 
 
Paediatric population 
 
Lojuxta has not been investigated in children less than 18 years of age. 
 
Elderly population 
 
Lojuxta has not been investigated in patients aged 65 years or older.  
 
5.3 Preclinical safety data 
 
In repeat-dose oral toxicology studies in rodents and dogs, the principal drug-related findings were 
lipid accumulation in the small intestine and/or liver associated with decreases in serum cholesterol 
and/or triglyceride levels. These changes are secondary to the mechanism of action of lomitapide. 
Other liver-related changes in repeat-dose toxicity studies in rats and dogs included increased serum 
aminotransferases, subacute inflammation (rats only), and single-cell necrosis. In a 1 year repeat-dose 
study in dogs there were no microscopic changes in the liver although serum AST was minimally 
increased in females.  
 
Pulmonary histiocytosis was observed in rodents. Decreased red blood cell parameters as well as 
poikilocytosis and/or anisocytosis were observed in dogs. Testicular toxicity was observed in dogs at 
205 times the human exposure (AUC) at 60 mg in a 6-month study. No adverse effects on the testes 
were observed in a 1-year study in dogs at 64 times the human exposure at 60 mg. 
 
In a dietary carcinogenicity study in mice, lomitapide was administered up to 104 weeks at doses 
ranging from 0.3 to 45 mg/kg/day. There were statistically significant increases in the incidences of 
liver adenoma and carcinoma at doses ≥1.5 mg/kg/day in males (≥ 2 times the human exposure at 
60 mg daily based on AUC) and ≥7.5 mg/kg/day in females (≥ 9 times the human exposure at 60 mg 
based on AUC). Incidences of small intestinal carcinoma and/or combined adenoma and carcinoma 
(rare tumours in mice) were significantly increased at doses ≥15 mg/kg/day in males (≥ 26 times the 
human exposure at 60 mg based on AUC) and at 15 mg/kg/day in females (22 times the human 
exposure at 60 mg based on AUC).  
 
In an oral carcinogenicity study in rats, lomitapide was administered up to 99 weeks at doses up to 
7.5 mg/kg/day in males and 2.0 mg/kg/day in females. Focal hepatic fibrosis was observed in males 
and females and hepatic cystic degeneration was observed in males only. In high-dose males, an 
increased incidence of pancreatic acinar cell adenoma was observed at an exposure 6 times that in 
humans at 60 mg based on AUC. 
 
Lomitapide was not mutagenic or genotoxic in a battery of in vitro and in vivo studies. 
 



21 

Lomitapide had no effect on reproductive function in female rats at doses up to 1 mg/kg or in male rats 
at doses up to 5 mg/kg. Systemic exposures to lomitapide at these doses were estimated to be 4 times 
(females) and 5 times (males) higher than the human exposure at 60 mg based on AUC. 
 
Lomitapide was teratogenic in rats in the absence of maternal toxicity at an exposure (AUC) estimated 
to be twice that in humans at 60 mg. There was no evidence of embryofoetal toxicity in rabbits at 
3 times the maximum recommended human dose (MRHD) of 60 mg based on body surface area. 
Embryofoetal toxicity was observed in rabbits in the absence of maternal toxicity at ≥6.5 times the 
MRHD. In ferrets, lomitapide was both maternally toxic and teratogenic at <1 times the MRHD. 
 
 
6. PHARMACEUTICAL PARTICULARS 
 
6.1 List of excipients 
 
Capsule content: 
 
Pregelatinised starch (maize) 
Sodium starch glycolate (Type A) 
Microcrystalline cellulose 
Lactose monohydrate 
Silica, colloidal anhydrous 
Magnesium stearate 
 
Capsule shell:  
 
Lojuxta 5 mg, 10 mg hard capsules 
Gelatin  
Titanium dioxide (E171) 
Red iron oxide (E172) 
 
Lojuxta 20 mg hard capsules 
Gelatin  
Titanium dioxide (E171) 
 
Lojuxta 30 mg hard capsules 
Gelatin  
Titanium dioxide (E171) 
Red iron oxide (E172) 
Yellow iron oxide (E172) 
 
Lojuxta 40 mg, 60 mg hard capsules 
Gelatin  
Titanium dioxide (E171) 
Yellow iron oxide (E172) 
 
Printing ink:  
 
Shellac 
Black iron oxide (E172) 
Propylene glycol  
 
6.2 Incompatibilities 
 
Not applicable. 
 



22 

6.3 Shelf life 
 
3 years. 
 
6.4 Special precautions for storage 
 
Store below 30°C. 
Keep the bottle tightly closed in order to protect from moisture. 
 
6.5 Nature and contents of container 
 
High density polyethylene (HDPE) bottle fitted with a polyester/aluminium foil/cardboard induction 
seal and polypropylene screw cap. 
 
Package sizes are:  
28 capsules 
 
6.6 Special precautions for disposal  
 
No special requirements. 
 
 
7. MARKETING AUTHORISATION HOLDER 
 
Amryt Pharmaceuticals DAC 
90 Harcourt Street 
Dublin 2 
Ireland 
 
 
8. MARKETING AUTHORISATION NUMBER(S)  
 
EU/1/13/851/001 – Lojuxta 5 mg hard capsules 
EU/1/13/851/002 – Lojuxta 10 mg hard capsules 
EU/1/13/851/003 – Lojuxta 20 mg hard capsules 
EU/1/13/851/004 – Lojuxta 30 mg hard capsules 
EU/1/13/851/005 – Lojuxta 40 mg hard capsules 
EU/1/13/851/006 – Lojuxta 60 mg hard capsules 
 
 
9. DATE OF FIRST AUTHORISATION/RENEWAL OF THE AUTHORISATION 
 
Date of first authorisation: 31 July 2013  
 
Date of latest renewal: 23 April 2018 
 
 
10. DATE OF REVISION OF THE TEXT 
 
Detailed information on this medicinal product is available on the website of the European Medicines 
Agency http://www.ema.europa.eu. 

http://www.ema.europa.eu/


23 

 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 

ANNEX II 
 
A. MANUFACTURER RESPONSIBLE FOR BATCH RELEASE  
 
B. CONDITIONS OR RESTRICTIONS REGARDING SUPPLY AND USE 
 
C. OTHER CONDITIONS AND REQUIREMENTS OF THE MARKETING 

AUTHORISATION 
 
D. CONDITIONS OR RESTRICTIONS WITH REGARD TO THE SAFE AND 

EFFECTIVE USE OF THE MEDICINAL PRODUCT 
 
E. SPECIFIC OBLIGATION TO COMPLETE POST-AUTHORISATION MEASURES 

FOR THE MARKETING AUTHORISATION UNDER EXCEPTIONAL 
CIRCUMSTANCES 

 
 

 



24 

A. MANUFACTURER RESPONSIBLE FOR BATCH RELEASE 
 
Name and address of the manufacturer responsible for batch release 
 
HÄLSA Pharma GmbH 
Hafenweg 18-20 
48155 Münster 
Germany 
 
 
B. CONDITIONS OR RESTRICTIONS REGARDING SUPPLY AND USE 
 
Medicinal product subject to restricted medical prescription (see Annex I: Summary of Product 
Characteristics, section 4.2). 
 
 
C. OTHER CONDITIONS AND REQUIREMENTS OF THE MARKETING 

AUTHORISATION  
 
• Periodic safety update reports  
 
The requirements for submission of periodic safety update reports for this medicinal product are set 
out in the list of Union reference dates (EURD list) provided for under Article 107c(7) of Directive 
2001/83/EC and any subsequent updates published on the European medicines web-portal. 
 
 
D. CONDITIONS OR RESTRICTIONS WITH REGARD TO THE SAFE AND 

EFFECTIVE USE OF THE MEDICINAL PRODUCT 
 
• Risk Management Plan (RMP) 
 
The MAH shall perform the required pharmacovigilance activities and interventions detailed in the 
agreed RMP presented in Module 1.8.2 of the Marketing Authorisation and any agreed subsequent 
updates of the RMP. 
 
An updated RMP should be submitted: 

• At the request of the European Medicines Agency; 
• Whenever the risk management system is modified, especially as the result of new 

information being received that may lead to a significant change to the benefit/risk profile or 
as the result of an important (pharmacovigilance or risk minimisation) milestone being 
reached.  

 
• Additional risk minimisation measures   
 
The MAH shall provide an educational pack prior to launch targeting all physicians who are 
expected to prescribe/use lomitapide.  
The physician educational pack should contain: 

• The Summary of Product Characteristics 
• The Prescriber Guide 
• Patient Brochures 
• Patient Alert Cards 

 
 
The MAH must agree the content and format of the educational materials together with a 
communication plan with the national competent authority in each Member State prior to distribution 
in their territory. 



25 

 
The Prescriber Guide shall include the following key elements: 
 
Appropriate patient selection 
 
• Lojuxta is only indicated for use in adult patients with HoFH;  
• The safety and effectiveness of Lojuxta in children below the age of 18 have not been established; 
• Treatment with Lojuxta should be initiated and monitored by a physician experienced in the 

treatment of lipid disorders; 
• That Lojuxta was teratogenic in non-clinical studies and that women of child-bearing potential 

must be non-pregnant and using effective contraception prior to initiating treatment.  
 
Gastrointestinal (GI) effects 
 
• Information on undesirable effects, including diarrhoea, nausea, flatulence, abdominal pain or 

discomfort, abdominal distension, vomiting, dyspepsia, eructation and decreased appetite; 
• Contraindication for use in patients with a known significant or chronic bowel disease such as 

inflammatory bowel disease or malabsorption; 
• Advice on escalating Lojuxta dose gradually to improve tolerability of the medicine; 
• Advice to patients about:  

 − The need to follow a low-fat diet (i.e. patients should follow a diet supplying less than 20% of 
energy from fat); 

 − The timing of medicine intake (Lojuxta should be taken on an empty stomach, at least 2 hours 
after the evening meal); 

 − The need to take daily dietary supplements (i.e. 400 IU vitamin E, approximately 200 mg 
linoleic acid, 110 mg eicosapentaenoic acid (EPA), 210 mg alpha linolenic acid (ALA) and 
80 mg docosahexaenoic acid (DHA) per day). 

 
Hepatic events related to elevated aminotransferases and progressive liver disease 
 
• Information about contraindication in patients with moderate or severe pre-existing hepatic 

impairment/disease, including those with unexplained persistent abnormal liver function tests;  
• Information about clinical findings (i.e., hepatic enzyme increases and steatosis) in subjects treated 

with Lojuxta during the developmental phase; 
• Advice to exercise caution if Lojuxta is used with other hepatotoxic medicinal products and to 

consider more frequent monitoring of liver-related tests; 
• Advice to patients about the risk of concomitant alcohol intake; 
• Advice on monitoring liver function (measuring hepatic enzymes and total bilirubin) before and 

during treatment with Lojuxta and routine screening to detect presence of steatohepatitis and 
hepatic fibrosis including specific details of the screening tests at baseline and annually as follows: 

 - Imaging for tissue elasticity, e.g. Fibroscan, acoustic radiation force impulse (ARFI), or 
magnetic resonance (MR) elastography; 

 - Measurement of biomarkers and/or scoring methods. This should include at least one marker 
in each of the following categories:  

 gamma-GT, serum albumin (liver injury); 
 high sensitivity C-reactive protein (hs-CRP), erythrocyte sedimentation rate 

(ESR), CK-18 Fragment, NashTest (liver inflammation); 
 Enhanced Liver Fibrosis (ELF) panel, Fibrometer, AST/ALT ratio, Fib-4 score, 

Fibrotest (liver fibrosis). 
 
Use in women of childbearing potential 
 
• That Lomitapide was teratogenic in non-clinical studies and is contraindicated in women who are 

or may become pregnant. Women who become pregnant should be counselled and referred to an 
expert in teratology; 

• Before initiating treatment in women of child-bearing potential:  



26 

 − The absence of pregnancy should be confirmed; 
 − Appropriate advice on effective methods of contraception should be provided, and effective 

contraception initiated;  
• Warning about possible loss of effectiveness of oral contraceptives due to diarrhoea or vomiting 

and need for additional contraception until 7 days after resolution of symptoms;  
• Women should tell their doctor immediately if they suspect that they might be pregnant. 
 
Drug interactions 
 
• Information about interactions with CYP3A4 inhibitors and inducers, coumarin anticoagulants, 

statins, P-gp substrates, oral contraceptives, bile acid sequestrants and grapefruit juice; 
• Importance of fatty acid and soluble vitamins supplementation; 
• Compliance with the supplementation regimen should be verified at regular scheduled 

appointments and the importance emphasised. 
 
Educational materials for patients 
 
Information that the educational materials for patients included in the prescribers pack can be used for 
patient counselling. 
A copy of the patient brochure and patient alert card shall be provided to all patients at the time 
Lojuxta treatment is initiated. 
Patients shall be informed of the necessity to carry the patient alert card with them and show it to all 
doctors that treat them.  
Lomitapide Observational Worldwide Evaluation Registry  
Information about the existence and importance of the registry aiming to systematically collect 
information on the safety and effectiveness outcomes of patients treated with lomitapide. 
Prescribers are encouraged to enrol all patients treated with Lojuxta into a global registry. 
 
Patient brochure 
 
The patient brochure shall include the following key elements: 
• Not to take Lojuxta if patient has liver problems, or unexplained abnormal liver tests; 
• Information that Lojuxta may cause liver problems;  
• The need to inform their doctor if they have had any liver problems in the past;  
• The need to inform their doctor of all other medicines they are taking as special care should be 

taken if other medicines which can cause liver problems are taken at the same time; 
• Symptoms of liver disease for which the patient should consult a doctor; 
• An explanation of the types of tests required (imaging and blood) to check liver function and the 

importance of them being performed regularly; 
• Information that Lojuxta was teratogenic in non-clinical studies and should not be taken during 

pregnancy or by patients trying to get pregnant;  
• Women of childbearing potential should have adequate birth control and should tell their doctors 

immediately if they suspect they may be pregnant;  
• Lojuxta may cause diarrhoea and vomiting and if it does, patients using oral contraception should 

use additional contraceptive methods for 7 days after symptoms have resolved; 
• Information about interactions with CYP3A4 inhibitors and inducers, coumarin anticoagulants, 

statins, P-gp substrates, oral contraceptives, bile acid sequestrants; 
• The need to avoid alcohol; 
• The need to avoid grapefruit juice; 
• Importance of fatty acid and fat soluble vitamin (Vitamin E) supplementation; 
• Information on the importance of following a low-fat diet (a diet supplying less than 20% of energy 

from fat);  
• Information about taking Lojuxta at bedtime with water at least 2 hours after the evening meal and 

without food; 
• Information about the existence and importance of the Lomitapide Observational Worldwide 

Evaluation Registry aiming to systematically collect information on the safety and effectiveness 



27 

outcomes of patients treated with lomitapide. 
 
Patient Alert card 
 
The purpose of the patient alert card is to inform health care professionals of potential drug-drug 
interactions before any additional medicinal product is prescribed. Patients will be instructed to carry 
this card and show it to all doctors who treat them. 
 
This card will give information about interactions with:  

o CYP 3A4 inhibitors  
o CYP 3A4 inducers  
o coumarin anticoagulants  
o statins  
o P-gp substrates  
o Oestrogen-containing oral contraceptives 

 
• Obligation to conduct post-authorisation measures  
 
The MAH shall complete, within the stated timeframe, the below measure: 
 
Description Due date 
CAPTURE study – effects of lomitapide on carotid and aortic 
atherosclerosis in patients treated with lomitapide in usual care.The 
applicant shall conduct a clinical study with adequate surrogate 
endpoints on vascular outcomes using imaging techniques to monitor 
vascular function, disease stabilisation and/or regression. 

The final study report shall 
be submitted by 
31 December 2021. 

 
 



28 

E. SPECIFIC OBLIGATION TO COMPLETE POST-AUTHORISATION MEASURES 
FOR THE MARKETING AUTHORISATION UNDER EXCEPTIONAL 
CIRCUMSTANCES  

 
This being an approval under exceptional circumstances and pursuant to Article 14(8) of Regulation 
(EC) No 726/2004, the MAH shall conduct, within the stated timeframe, the following measure: 

Description Due date 
LOWER registry – long term prospective observational study to 
systematically collect information on the safety and effectiveness 
outcomes of patients treated with lomitapide. 
PER - Pregnancy exposure registry – to evaluate the occurrence and 
outcomes of pregnancy in females of reproductive potential treated with 
lomitapide who decide to continue the pregnancy following advice from 
a teratologist. 
The applicant shall set up a long term prospective observational study to 
systematically collect information on the safety and effectiveness 
outcomes of patients treated with lomitapide.  

 
The objectives of the study are: 
• To evaluate the occurrence of the following in patients treated with 
lomitapide: 

o Hepatic events 
o Gastrointestinal events 
o Small bowel, hepatic, colorectal and pancreatic tumours  
o Events associated with coagulopathy  
o Major Adverse Cardiovascular Events (MACE) events  
o Death, including cause of death 

• To evaluate the occurrence and outcomes of pregnancy in females of 
reproductive potential treated with lomitapide who decide to continue 
the pregnancy following advice from a teratologist.  
• To evaluate the long-term effectiveness of lomitapide in maintaining 
control of serum lipid levels in clinical practice. 
• To evaluate whether prescribers of lomitapide are following the 
screening and monitoring recommendations as specified in the product 
information and the educational materials. 
 

Annual reports will be 
submitted at time of annual 
reassessment 
 

 



29 

 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 

ANNEX III 
 

LABELLING AND PACKAGE LEAFLET 



30 

 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 

A. LABELLING 
 



31 

 
PARTICULARS TO APPEAR ON THE OUTER PACKAGING AND THE IMMEDIATE 
PACKAGING 
 
CARTON AND BOTTLE (5 mg, 10 mg, 20 mg, 30 mg, 40 mg and 60 mg) 
 
 
1. NAME OF THE MEDICINAL PRODUCT 
 
Lojuxta 5 mg hard capsules 
Lojuxta 10 mg hard capsules 
Lojuxta 20 mg hard capsules 
Lojuxta 30 mg hard capsules 
Lojuxta 40 mg hard capsules 
Lojuxta 60 mg hard capsules 
lomitapide 
 
 
2. STATEMENT OF ACTIVE SUBSTANCE(S) 
 
Each hard capsule contains lomitapide mesylate equivalent to 5 mg lomitapide. 
Each hard capsule contains lomitapide mesylate equivalent to 10 mg lomitapide. 
Each hard capsule contains lomitapide mesylate equivalent to 20 mg lomitapide. 
Each hard capsule contains lomitapide mesylate equivalent to 30 mg lomitapide. 
Each hard capsule contains lomitapide mesylate equivalent to 40 mg lomitapide. 
Each hard capsule contains lomitapide mesylate equivalent to 60 mg lomitapide. 
 
 
3. LIST OF EXCIPIENTS 
 
Contains lactose. 
See package leaflet for further information. 
 
 
4. PHARMACEUTICAL FORM AND CONTENTS 
 
28 hard capsules 
 
 
5. METHOD AND ROUTE(S) OF ADMINISTRATION 
 
Read the package leaflet before use. 
Oral use 
 
 
6. SPECIAL WARNING THAT THE MEDICINAL PRODUCT MUST BE STORED OUT 

OF THE SIGHT AND REACH OF CHILDREN 
 
Keep out of the sight and reach of children. 
 
 
7. OTHER SPECIAL WARNING(S), IF NECESSARY 
 
 



32 

8. EXPIRY DATE 
 
EXP  
 
 
9. SPECIAL STORAGE CONDITIONS 
 
Store below 30°C.  
Keep the bottle tightly closed in order to protect from moisture. 
 
 
10. SPECIAL PRECAUTIONS FOR DISPOSAL OF UNUSED MEDICINAL PRODUCTS 

OR WASTE MATERIALS DERIVED FROM SUCH MEDICINAL PRODUCTS, IF 
APPROPRIATE 

 
 
11. NAME AND ADDRESS OF THE MARKETING AUTHORISATION HOLDER 
 
Amryt Pharmaceuticals DAC 
90 Harcourt Street 
Dublin 2 
Ireland 
 
 
12. MARKETING AUTHORISATION NUMBER(S)  
 
EU/1/13/851/001 
EU/1/13/851/002 
EU/1/13/851/003 
EU/1/13/851/004 
EU/1/13/851/005 
EU/1/13/851/006 
 
 
13. BATCH NUMBER 
 
Lot 
 
 
14. GENERAL CLASSIFICATION FOR SUPPLY 
 
 
 
15. INSTRUCTIONS ON USE 
 
 



33 

16. INFORMATION IN BRAILLE 
 
lojuxta 5 mg 
lojuxta 10 mg 
lojuxta 20 mg 
lojuxta 30 mg 
lojuxta 40 mg 
lojuxta 60 mg 
 
 
17. UNIQUE IDENTIFIER – 2D BARCODE 
 
2D barcode carrying the unique identifier included. 
 
 
18. UNIQUE IDENTIFIER - HUMAN READABLE DATA 
 
PC: {number}  
SN: {number} 
NN: {number} 
 
 
 



34 

 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 

B. PACKAGE LEAFLET 



35 

Package leaflet: Information for the user 
  

Lojuxta 5 mg hard capsules 
Lojuxta 10 mg hard capsules 
Lojuxta 20 mg hard capsules 

lomitapide  
 

 
This medicine is subject to additional monitoring. This will allow quick identification of new 

safety information. You can help by reporting any side effects you may get. See the end of section 4 
for how to report side effects. 
 
Read all of this leaflet carefully before you start taking this medicine because it contains 
important information for you. 
- Keep this leaflet. You may need to read it again. 
- If you have any further questions, ask your doctor or pharmacist. 
- This medicine has been prescribed for you only. Do not pass it on to others. It may harm them, 

even if their signs of illness are the same as yours. 
- If you get any side effects, talk to your doctor or pharmacist. This includes any possible side 

effects not listed in this leaflet. See section 4. 
 
What is in this leaflet 
 
1. What Lojuxta is and what it is used for 
2. What you need to know before you take Lojuxta 
3. How to take Lojuxta 
4. Possible side effects 
5. How to store Lojuxta 
6. Contents of the pack and other information 
 
 
1. What Lojuxta is and what it is used for 
 
Lojuxta contains the active substance called lomitapide. Lomitapide is a “lipid-modifying agent” 
which works by blocking the action of “microsomal triglyceride transfer protein.” This protein is 
located within the liver and the gut cells, where it is involved in assembling fatty substances into larger 
particles that are then released into the blood stream. By blocking this protein, the medicine decreases 
the level of fats and cholesterol (lipids) in the blood. 
 
Lojuxta is used to treat adult patients with very high cholesterol because of a condition that runs in 
their families (homozygous familial hypercholesterolaemia or HoFH). It is typically passed down by 
both father and mother, who also have high cholesterol passed down from their parents. The patient’s 
“bad” cholesterol level is very high from a very early age. The “bad” cholesterol can lead to heart 
attacks, strokes or other events at an early age. Lojuxta is used with a low-fat diet and other lipid 
lowering treatments to decrease your cholesterol levels. 
 
Lojuxta can lower blood levels of: 

• low density lipoprotein (LDL) cholesterol (“bad” cholesterol) 
• total cholesterol 
• apolipoprotein-B, a protein that carries “bad cholesterol” in the blood 
• triglycerides (fat carried in the blood)  

 
 



36 

2. What you need to know before you take Lojuxta 
 
Do not take Lojuxta: 
• if you are allergic to lomitapide or any of the other ingredients of this medicine (listed in section 

6). 
• if you have liver problems or unexplained abnormal liver tests.  
• if you have bowel problems or cannot absorb food from your bowel. 
• if you take more than 40 mg of simvastatin daily (another medicine used to lower cholesterol). 
• if you take any of these medicines that affect the way lomitapide is broken down in the body: 

o itraconazole, ketoconazole, fluconazole, voriconazole, posaconazole (for fungal 
infections) ; 

o telithromycin, clarithromycin, erythromycin (for bacterial infections) 
o indinavir, nelfinavir, saquinavir (for HIV infection) ; 
o diltiazem, verapamil (for high blood pressure, or angina), and dronedarone (to 

regulate heart rhythm). 
• if you are pregnant, trying to get pregnant, or think you may be pregnant (see section 2 under 

‘Pregnancy and breast-feeding’). 
 
Warnings and precautions  
Talk to your doctor or pharmacist before taking Lojuxta if you:  
 
• have had liver problems, including liver problems whilst taking other medicines. 

These capsules may cause side effects which can also be symptoms of liver problems. These 
side-effects are listed in section 4 and you must tell your doctor immediately if you have any 
of these signs and symptoms, as they may be caused by liver damage.  Your doctor will give 
you a blood test to check your liver before you start taking these capsules, if your dose is 
increased, and regularly during treatment. These blood tests help your doctor adjust your dose. 
If your tests show some liver problems, your doctor may decide to reduce your dose or stop the 
treatment. 

 
You may in some cases experience loss of fluids/dehydration, e.g. in case of vomiting, nausea and 
diarrhoea. It is important to avoid dehydration by drinking enough fluids (see section 4). 
 
Children and adolescents 
No studies have been conducted in children and adolescents under the age of 18. Therefore the use of 
this medicine in children and adolescents is not recommended. 
 
Other medicines and Lojuxta 
Tell your doctor or pharmacist if you are taking, have recently taken, or might take any other 
medicines. 
 
Other medicines may affect the way Lojuxta works. Do not take any of the following medicines with 
Lojuxta:  

• some medicines for bacterial, fungal or HIV infection (see section 2 under ‘Do not take 
Lojuxta’) 

• some medicines for high blood pressure, angina or to regulate heart rhythm (see section 2 under: 
‘Do not take Lojuxta’) 

 
You must also tell your doctor or pharmacist if you are taking any of the following medicines, as they 
may need to change your dose of Lojuxta: 

• medicines which lower cholesterol (e.g. atorvastatin) 
• combined oral contraceptives (e.g. ethinylestradiol, norgestimate) 
• glucocorticoids (e.g. beclometasone, prednisolone) steroid medicines used to treat inflammation 

in conditions such as severe asthma, arthritis 
• medicines to treat cancer (e.g. bicalutamide, lapatinib, methotrexate, nilotinib, pazopanib, 

tamoxifen) or nausea/vomiting with cancer treatment (e.g. fosaprepitant) 



37 

• medicines to reduce the activity of the immune system (e.g. ciclosporin, tacrolimus) 
• medicines to treat bacterial or fungal infections (e.g. nafcillin, azithromycin, roxithromycin, 

clotrimazole) 
• medicines to treat and prevent blood clots (e.g. cilostazol, ticagrelor) 
• medicines to treat angina – chest pain caused by the heart (e.g. ranolazine) 
• medicines to reduce blood pressure (e.g. amlodipine, lacidipine) 
• medicines to regulate heart rhythm (e.g. amiodarone) 
• medicines to treat epilepsy (e.g. phenobarbital, carbamazepine, phenytoin) 
• medicines to treat diabetes (e.g. pioglitazone, linagliptin) 
• medicines to treat tuberculosis (e.g. isoniazid, rifampicin) 
• tetracycline antibiotics to treat infections such as urinary tract infections 
• medicines to treat anxiety disorders and depression (e.g. alprazolam, fluoxetine, fluvoxamine) 
• antacids (e.g. ranitidine, cimetidine) 
• aminoglutethimide – a medicine used to treat Cushing’s syndrome 
• medicines to treat severe acne (e.g. isotretinoin) 
• paracetamol – to treat pain  
• medicines to treat cystic fybrosis (e.g. ivacaftor) 
• medicines to treat urinary incontinence (e.g. propiverine) 
• medicines to treat low levels of sodium in the blood (e.g. tolvaptan) 
• medicines to treat excessive daytime sleepiness (e.g. modafinil)  
• some herbal medicines: 

o St. John’s Wort (for depression) 
o Ginkgo (to improve memory) 
o Goldenseal (for inflammation and infection) 

 
Lojuxta may affect the way other medicines work. Tell your doctor or pharmacist if you are taking any 
of the following medicines: 

• oral contraceptives (see section 2 under ‘Pregnancy and breast-feeding’)  
• other medicines used to lower cholesterol such as: 

o statins such as simvastatin. The risk of liver damage is increased when this medicine 
is used at the same time as statins. Muscle aches and pains (myalgia) or weakness 
(myopathy) may also occur. Contact your doctor immediately if you experience 
any unexplained muscle aches and pains, tenderness or weakness. You should not 
take more than 40 mg of simvastatin when using Lojuxta (see section 2 under ‘Do not 
take Lojuxta’)  

• coumarin anticoagulants for thinning the blood ( e.g. warfarin) 
• medicines to treat cancer (e.g. everolimus, imatinib, lapatinib, nilotinib, topotecan) 
• medicines to reduce the activity of the immune system (e.g. sirolimus) 
• medicines to treat HIV (e.g. maraviroc) 
• medicines to treat and prevent blood clots (e.g. dabigatran etexilate) 
• medicines to treat angina – chest pain caused by the heart (e.g. ranolazine) 
• medicines to reduce blood pressure (e.g. talinolol, aliskiren, ambrisentan) 
• medicines to regulate heart rhythm (e.g. digoxin) 
• medicines to treat diabetes (e.g. saxagliptin, sitagliptin) 
• medicines to treat gout (e.g. colchicine) 
• medicines to treat low blood sodium level (e.g. tolvaptan) 
• anti-histamine medicines to treat hayfever (e.g. fexofenadine) 

  
Lojuxta with food, drink and alcohol 

• Do not drink any type of grapefruit juice. 
• The use of alcohol during Lojuxta treatment is not recommended. 
• Your dose of Lojuxta may need to be adjusted if you consume peppermint oil or Seville 

oranges. 
• To lower the chance of stomach problems, you must stay on a low-fat diet whilst taking this 

medicine. Work with a dietitian to learn what you can eat while taking Lojuxta. 



38 

  
Pregnancy and breast-feeding 
Do not take this medicine if you are pregnant, trying to get pregnant, or think you may be pregnant, as 
there is a possibility that it could harm an unborn baby. If you get pregnant while taking this medicine, 
call your doctor immediately and stop taking the capsules. 
 
Pregnancy 

• Before starting treatment you should confirm you are not pregnant and are using effective 
contraception, as advised by your doctor. If you use contraceptive pills and suffer from an 
episode of diarrhoea or vomiting that lasts more than 2 days, you must use an alternative 
method of contraception (e.g. condoms, diaphragm) for 7 days following resolution of 
symptoms. 

• If, after you have started treatment with Lojuxta, you decide that you would like to become 
pregnant, please inform your doctor, as your treatment will need to be changed. 

 
Breast-feeding 

• It is not known if Lojuxta is passed into breast milk. Please tell your doctor if you are breast-
feeding or planning to breast-feed. Your doctor may advise you to stop taking Lojuxta or to 
stop breast-feeding. 

 
Driving and using machines 
Your treatment may affect your ability to drive or use machines. If you feel dizzy during treatment 
then do not drive or use machines until you feel better. 
 
Lojuxta contains lactose 
If you have been told by your doctor that you have an intolerance to some sugars, contact your doctor 
before taking this medicine. 
 
 
3. How to take Lojuxta 
 
Always take this medicine exactly as your doctor or pharmacist has told you. Check with your doctor 
or pharmacist if you are not sure. These capsules should be given to you by a doctor experienced in 
the treatment of lipid disorders who will also monitor you regularly. 
 
The recommended starting dose is a 5 mg capsule each day. Your doctor may increase your dose 
slowly over time, up to a maximum of 60 mg each day. Your doctor will tell you: 

• what dose to take and for how long. 
• when to increase or decrease your dose. 

Do not change the dose yourself. 
 

• Take this medicine once a day at bedtime with a glass of water at least 2 hours after your 
evening meal (see section 2 under ‘Lojuxta with food, drink and alcohol’).  

• Do not take this medicine with food, as taking these capsules with food can cause stomach 
problems.   

• If you take another medicine that lowers cholesterol by binding bile acids, such as colesevelam 
or cholestyramine, take the medicine that binds bile acids at least 4 hours before or 4 hours 
after you take Lojuxta.  

 
Because of the possibility of interactions with other medicines, your doctor may change the time of 
day you take your medicines. Alternatively, your doctor may decrease your dose of Lojuxta. Inform 
your doctor of any change in the medicines you are taking. 
 
You also need to take daily vitamin E and essential fatty acid (omega-3 and omega-6) supplements 
while taking this medicine. The usual dose that you will need to take is listed below. Ask your doctor, 
or dietitian how to obtain these supplements. See section 2 under ‘Lojuxta with food, drink and 
alcohol’. 



39 

 
Daily Amount  
Vitamin E  400 IU* 
Omega-3  Approximately  
EPA  110 mg* 
DHA 80 mg 
ALA 210 mg 
Omega-6   
Linoleic acid 200 mg  
* IU – international units, mg - milligrams 

 
If you take more Lojuxta than you should  
Contact your doctor or pharmacist immediately.  
 
If you forget to take Lojuxta 
Just take your normal dose at the usual time the next day. Do not take a double dose to make up for a 
forgotten dose.  
 
If you stop taking Lojuxta 
If you stop taking this medicine your cholesterol may rise again. You should contact your doctor 
before you stop taking this medicine. 
 
If you have any further questions on the use of this medicine, ask your doctor or pharmacist. 
 
 
4. Possible side effects 
 
Like all medicines, this medicine can cause side effects, although not everybody gets them. 
 
Serious side effects: 

• abnormal blood tests for liver function have been reported commonly (may affect up to 1 in 
10 people). The signs and symptoms of liver problems include: 

o nausea (feeling sick) 
o vomiting (being sick) 
o stomach pain 
o muscle aches and pains 
o fever 
o skin or whites of your eyes turn yellow 
o being more tired than usual 
o feeling like you have the flu 

 
Tell your doctor immediately if you have any of these symptoms as your doctor may decide to stop 
the treatment. 
 
The following other side effects have also occurred: 
 
Very common (may affect more than 1 in 10 people):  

• diarrhoea 
• nausea and vomiting (feeling or being sick) 
• stomach pain, discomfort or stomach bloating 
• decreased appetite 
• indigestion 
• flatulence (wind) 
• constipation  
• weight loss 

 



40 

Common (may affect up to 1 in 10 people):  
• inflammation of the stomach and intestine that causes diarrhoea and vomiting  
• regurgitation (bringing food back up) 
• burping 
• feeling of incomplete defaecation (bowel movement), urgent need to defaecate 
• bleeding from your rectum (back passage) or blood in your stool  
• dizziness, headache, migraine 
• tiredness, lack of energy or general weakness 
• enlarged, damaged or fatty liver 
• purple discoloration of the skin, solid bumps on the skin, rash, yellow bumps on the skin 
• changes to blood clotting tests 
• changes to blood cell count  
• decrease in levels of potassium, carotene, vitamin E, vitamin K in your blood 
• muscle spasms 
 

Uncommon (may affect up to 1 in 100 people):  
• flu or cold, fever, inflammation of your sinuses, cough 
• low red blood cell count (anaemia) 
• dehydration, dry mouth 
• increased appetite 
• burning or prickling of the skin 
• swelling of the eye 
• ulcer or sore spot in the throat 
• vomiting blood 
• dry skin 
• blister 
• excessive sweating 
• joint pain or swelling, pain in hands or feet 
• muscle pain 
• blood or protein in the urine 
• chest pain 
• changes to your walking (gait)  
• abnormal lung function test 

 
Not known (frequency cannot be estimated from the available data) 

• hair loss (alopecia) 
• muscle pain (myalgia) 
• loss of fluid that may cause headache, dry mouth, dizziness, tiredness or unconsciousness 

(dehydration) 
 
Reporting of side effects 
If you get any side effects, talk to your doctor or pharmacist. This includes any possible side effects 
not listed in this leaflet. You can also report side effects directly via the national reporting system 
listed in Appendix V. By reporting side effects you can help provide more information on the safety of 
this medicine. 
 
 
5. How to store Lojuxta 
 
Keep this medicine out of the sight and reach of children. 
 
Do not use this medicine after the expiry date which is stated on the label or carton after “EXP”. The 
expiry date refers to the last day of that month. 
 

http://www.ema.europa.eu/docs/en_GB/document_library/Template_or_form/2013/03/WC500139752.doc


41 

Store below 30°C.  
Keep the bottle tightly closed in order to protect from moisture. 
 
Do not throw away any medicines via wastewater or household waste. Ask your pharmacist how to 
throw away medicines you no longer use. These measures will help to protect the environment. 
 
 
6. Contents of the pack and other information 
 
What Lojuxta contains  

• The active substance is lomitapide.  
Lojuxta 5 mg: each hard capsule contains lomitapide mesylate equivalent to 5 mg lomitapide. 
Lojuxta 10 mg: each hard capsule contains lomitapide mesylate equivalent to 10 mg lomitapide. 
Lojuxta 20 mg: each hard capsule contains lomitapide mesylate equivalent to 20 mg lomitapide. 

• The other ingredients are: pregelatinized starch, sodium starch glycolate (Type A), 
microcrystalline cellulose, lactose monohydrate, silica colloidal anhydrous and magnesium 
stearate (for information on lactose monohydrate, see section 2 under ‘Lojuxta contains 
lactose’).  

 
Capsule shell: 
• The capsule shell for the 5 mg and 10 mg capsules contains gelatin, titanium dioxide (E171) and 

red iron oxide (E172). 
• The capsule shell for the 20 mg capsule contains gelatin and titanium dioxide (E171). 
• All capsules have edible black ink for printing. 

 
What Lojuxta looks like and contents of the pack 

• Lojuxta 5 mg is an orange cap/orange body hard capsule with “5 mg” printed on the body and 
“A733“ printed on the cap in black ink. 

• Lojuxta 10 mg is an orange cap/white body hard capsule with “10 mg” printed on the body and 
“A733“ printed on the cap in black ink.  

• Lojuxta 20 mg is a white cap/white body hard capsule with “20 mg” printed on the body and 
“A733” printed on the cap in black ink. 
 

Pack sizes are:  
28 capsules. 
 
Marketing Authorisation Holder 
Amryt Pharmaceuticals DAC 
90 Harcourt Street 
Dublin 2 
Ireland 
 
Manufacturer 
HÄLSA Pharma GmbH 
Hafenweg 18-20 
48155 Münster 
Germany 
 



42 

For any information about this medicine, please contact the local representative of the Marketing 
Authorisation Holder: 
 
België/Belgique/Belgien 
Amryt Pharmaceuticals DAC 
Tél/Tel: +800 44 474447  
Tél/Tel: +44 1604 549952 
medinfo@amrytpharma.com 
 

Lietuva 
Amryt Pharmaceuticals DAC 
Tel: +800 44 474447 
Tel: +44 1604 549952 
medinfo@amrytpharma.com 

България 
Amryt Pharmaceuticals DAC 
Teл.: +44 1604 549952 
medinfo@amrytpharma.com 
 

Luxembourg/Luxemburg 
Amryt Pharmaceuticals DAC 
Tel: +800 44 474447 
Tel: +44 1604 549952 
medinfo@amrytpharma.com 
 

Česká republika 
Amryt Pharmaceuticals DAC 
Tel: +800 44 474447  
Tel: +44 1604 549952 
medinfo@amrytpharma.com 
 

Magyarország 
Amryt Pharmaceuticals DAC 
Tel: +800 44 474447  
Tel: +44 1604 549952 
medinfo@amrytpharma.com 

Danmark 
Amryt Pharmaceuticals DAC 
Tlf: +800 44 474447 
Tlf: +44 1604 549952 
medinfo@amrytpharma.com 
 

Malta 
Amryt Pharmaceuticals DAC 
Tel: +44 1604 549952 
medinfo@amrytpharma.com 

Deutschland 
Amryt Pharmaceuticals DAC 
Tel: +800 44 474447  
Tel: +44 1604 549952 
medinfo@amrytpharma.com 
 

Nederland 
Amryt Pharmaceuticals DAC 
Tel: +800 44 474447  
Tel: +44 1604 549952 
medinfo@amrytpharma.com 

Eesti 
Amryt Pharmaceuticals DAC 
Tel: +800 44 474447  
Tel: +44 1604 549952 
medinfo@amrytpharma.com 
 

Norge 
Amryt Pharmaceuticals DAC 
Tlf: +44 1604 549952 
medinfo@amrytpharma.com 

Ελλάδα 
Amryt Pharmaceuticals DAC 
Tηλ: +800 44 474447  
Tηλ: +44 1604 549952 
medinfo@amrytpharma.com 
 

Österreich 
Amryt Pharmaceuticals DAC 
Tel: +800 44 474447 
Tel: +44 1604 549952 
medinfo@amrytpharma.com 
 

España 
Amryt Pharmaceuticals DAC 
Tel: +800 44 474447 
Tel: +44 1604 549952 
medinfo@amrytpharma.com 
 

Polska 
Amryt Pharmaceuticals DAC 
Tel: +800 44 474447  
Tel: +44 1604 549952 
medinfo@amrytpharma.com 

France 
Amryt Pharmaceuticals DAC 
Tél: +800 44 474447  
Tel: +44 1604 549952 
medinfo@amrytpharma.com 
 

Portugal 
Amryt Pharmaceuticals DAC 
Tel: +800 44 474447 
Tel: +44 1604 549952 
medinfo@amrytpharma.com  
 

mailto:medinfo@amrytpharma.com
mailto:medinfo@amrytpharma.com
mailto:medinfo@amrytpharma.com
mailto:medinfo@amrytpharma.com
mailto:medinfo@amrytpharma.com
mailto:medinfo@amrytpharma.com
mailto:medinfo@amrytpharma.com
mailto:medinfo@amrytpharma.com
mailto:medinfo@amrytpharma.com
mailto:medinfo@amrytpharma.com
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mailto:medinfo@amrytpharma.com
mailto:medinfo@amrytpharma.com
mailto:medinfo@amrytpharma.com
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mailto:medinfo@amrytpharma.com


43 

Hrvatska 
Amryt Pharmaceuticals DAC 
Tel: +44 1604 549952 
medinfo@amrytpharma.com 
 

România 
Amryt Pharmaceuticals DAC 
Tel: +44 1604 549952 
medinfo@amrytpharma.com 
 

Ireland 
Amryt Pharmaceuticals DAC 
Tel: +800 44 474447  
Tel: +44 1604 549952 
medinfo@amrytpharma.com 
 

Slovenija 
Amryt Pharmaceuticals DAC 
Tel: +44 1604 549952 
medinfo@amrytpharma.com 

Ísland 
Amryt Pharmaceuticals DAC 
Sími: +44 1604 549952 
medinfo@amrytpharma.com 
 

Slovenská republika 
Amryt Pharmaceuticals DAC 
Tel: +44 1604 549952 
medinfo@amrytpharma.com 

Italia 
Amryt Pharma Italy SRL 
Tel: +800 44 474447 
Tel: +44 1604 549952 
medinfo@amrytpharma.com 
 

Suomi/Finland 
Amryt Pharmaceuticals DAC 
Tel: +800 44 474447  
Tel: +44 1604 549952 
medinfo@amrytpharma.com 

Κύπρος 
Amryt Pharmaceuticals DAC 
Tηλ: +800 44 474447  
Tηλ: +44 1604 549952 
medinfo@amrytpharma.com 
 

Sverige 
Amryt Pharmaceuticals DAC 
Tel: +44 1604 549952 
medinfo@amrytpharma.com 
 

Latvija 
Amryt Pharmaceuticals DAC 
Tel: +44 1604 549952 
medinfo@amrytpharma.com 
 

United Kingdom 
Amryt Pharmaceuticals DAC 
Tel: +800 44 474447 (freephone) 
Tel: +44 1604 549952 
medinfo@amrytpharma.com 

 
 
This leaflet was last revised in > 
 
Other sources of information 
This medicine has been authorised under ‘exceptional circumstances’. This means that because of the 
rarity of this disease it has been impossible to get complete information on this medicine. The 
European Medicines Agency will review any new information on this medicine every year and this 
leaflet will be updated as necessary. 
 
Detailed information on this medicine is available on the European Medicines Agency web site: 
http://www.ema.europa.eu.  
 

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http://www.ema.europa.eu/


44 

Package leaflet: Information for the user 
  

Lojuxta 30 mg hard capsules 
Lojuxta 40 mg hard capsules 
Lojuxta 60 mg hard capsules 

lomitapide  
 

 
This medicine is subject to additional monitoring. This will allow quick identification of new 

safety information. You can help by reporting any side effects you may get. See the end of section 4 
for how to report side effects. 
 
Read all of this leaflet carefully before you start taking this medicine because it contains 

important information for you. 
- Keep this leaflet. You may need to read it again. 
- If you have any further questions, ask your doctor or pharmacist. 
- This medicine has been prescribed for you only. Do not pass it on to others. It may harm them, 

even if their signs of illness are the same as yours. 
- If you get any side effects, talk to your doctor or pharmacist. This includes any possible side 

effects not listed in this leaflet. See section 4. 
 
What is in this leaflet 
 
1. What Lojuxta is and what it is used for 
2. What you need to know before you take Lojuxta 
3. How to take Lojuxta 
4. Possible side effects 
5. How to store Lojuxta 
6. Contents of the pack and other information 
 
 
1. What Lojuxta is and what it is used for 
 
Lojuxta contains the active substance called lomitapide. Lomitapide is a “lipid-modifying agent” 
which works by blocking the action of “microsomal triglyceride transfer protein.” This protein is 
located within the liver and the gut cells, where it is involved in assembling fatty substances into larger 
particles that are then released into the blood stream. By blocking this protein, the medicine decreases 
the level of fats and cholesterol (lipids) in the blood. 
 
Lojuxta is used to treat adult patients with very high cholesterol because of a condition that runs in 
their families (homozygous familial hypercholesterolaemia or HoFH). It is typically passed down by 
both father and mother, who also have high cholesterol passed down from their parents. The patient’s 
“bad” cholesterol level is very high from a very early age. The “bad” cholesterol can lead to heart 
attacks, strokes or other events at an early age. Lojuxta is used with a low-fat diet and other lipid 
lowering treatments to decrease your cholesterol levels. 
 
Lojuxta can lower blood levels of: 

• low density lipoprotein (LDL) cholesterol (“bad” cholesterol) 
• total cholesterol 
• apolipoprotein-B, a protein that carries “bad cholesterol” in the blood 
• triglycerides (fat carried in the blood)  

 
 



45 

2. What you need to know before you take Lojuxta 
 
Do not take Lojuxta: 
• if you are allergic to lomitapide or any of the other ingredients of this medicine (listed in section 

6). 
• if you have liver problems or unexplained abnormal liver tests.  
• if you have bowel problems or cannot absorb food from your bowel. 
• if you take more than 40 mg of simvastatin daily (another medicine used to lower cholesterol). 
• if you take any of these medicines that affect the way lomitapide is broken down in the body: 

o itraconazole, ketoconazole, fluconazole, voriconazole, posaconazole (for fungal 
infections) ; 

o telithromycin, clarithromycin, erythromycin (for bacterial infections); 
o indinavir, nelfinavir, saquinavir (for HIV infection) ; 
o diltiazem, verapamil (for high blood pressure, or angina), and dronedarone (to 

regulate heart rhythm). 
• if you are pregnant, trying to get pregnant, or think you may be pregnant (see section 2 under 

‘Pregnancy and breast-feeding’). 
 
Warnings and precautions  
Talk to your doctor or pharmacist before taking Lojuxta if you:  
 
• have had liver problems, including liver problems whilst taking other medicines. 

These capsules may cause side effects which can also be symptoms of liver problems. These 
side-effects are listed in section 4 and you must tell your doctor immediately if you have any 
of these signs and symptoms, as they may be caused by liver damage. Your doctor will give you 
a blood test to check your liver before you start taking these capsules, if your dose is increased, 
and regularly during treatment. These blood tests help your doctor adjust your dose. If your tests 
show some liver problems, your doctor may decide to reduce your dose or stop the treatment. 

 
You may in some cases experience loss of fluids/dehydration, e.g. in case of vomiting, nausea and 
diarrhoea. It is important to avoid dehydration by drinking enough fluids (see section 4). 
 
Children and adolescents 
No studies have been conducted in children and adolescents under the age of 18. Therefore the use of 
this medicine in children and adolescents is not recommended. 
 
Other medicines and Lojuxta 
Tell your doctor or pharmacist if you are taking, have recently taken, or might take any other 
medicines. 
 
Other medicines may affect the way Lojuxta works. Do not take any of the following medicines with 
Lojuxta:  

• some medicines for bacterial, fungal or HIV infection (see section 2 under ‘Do not take 
Lojuxta’) 

• some medicines for high blood pressure, angina or to regulate heart rhythm (see section 2 under 
‘Do not take Lojuxta’) 

 
You must also tell your doctor or pharmacist if you are taking any of the following medicines, as they 
may need to change your dose of Lojuxta: 

• medicines which lower cholesterol (e.g. atorvastatin) 
• combined oral contraceptives (e.g. ethinylestradiol, norgestimate) 
• glucocorticoids (e.g. beclometasone, prednisolone) steroid medicines used to treat inflammation 

in conditions such as severe asthma, arthritis 
• medicines to treat cancer (e.g. bicalutamide, lapatinib, methotrexate, nilotinib, pazopanib, 

tamoxifen) or nausea/vomiting with cancer treatment (e.g. fosaprepitant) 
• medicines to reduce the activity of the immune system (e.g. ciclosporin, tacrolimus) 



46 

• medicines to treat bacterial or fungal infections (e.g. nafcillin, azithromycin, roxithromycin, 
clotrimazole) 

• medicines to treat and prevent blood clots (e.g. cilostazol, ticagrelor) 
• medicines to treat angina – chest pain caused by the heart (e.g. ranolazine) 
• medicines to reduce blood pressure (e.g. amlodipine, lacidipine) 
• medicines to regulate heart rhythm (e.g. amiodarone) 
• medicines to treat epilepsy (e.g. phenobarbital, carbamazepine, phenytoin) 
• medicines to treat diabetes (e.g. pioglitazone, linagliptin) 
• medicines to treat tuberculosis (e.g. isoniazid, rifampicin) 
• tetracycline antibiotics to treat infections such as urinary tract infections 
• medicines to treat anxiety disorders and depression (e.g. alprazolam, fluoxetine, fluvoxamine) 
• antacids (e.g. ranitidine, cimetidine) 
• aminoglutethimide – a medicine used to treat Cushing’s syndrome 
• medicines to treat severe acne (e.g. isotretinoin) 
• paracetamol – to treat pain  
• medicines to treat cystic fybrosis (e.g. ivacaftor) 
• medicines to treat urinary incontinence (e.g. propiverine) 
• medicines to treat low levels of sodium in the blood (e.g. tolvaptan) 
• medicines to treat excessive daytime sleepiness (e.g. modafinil)  
• some herbal medicines: 

o St. John’s Wort (for depression) 
o Ginkgo (to improve memory) 
o Goldenseal (for inflammation and infection) 

 
Lojuxta may affect the way other medicines work. Tell your doctor or pharmacist if you are taking any 
of the following medicines: 

• oral contraceptives (see section 2 under ‘Pregnancy and breast-feeding’)  
• other medicines used to lower cholesterol such as: 

o statins such as simvastatin. The risk of liver damage is increased when this medicine 
is used at the same time as statins. Muscle aches and pains (myalgia) or weakness 
(myopathy) may also occur. Contact your doctor immediately if you experience 
any unexplained muscle aches and pains, tenderness or weakness. You should not 
take more than 40 mg of simvastatin when using Lojuxta (see section 2 under ‘Do not 
take Lojuxta’)  

• coumarin anticoagulants for thinning the blood ( e.g. warfarin) 
• medicines to treat cancer (e.g. everolimus, imatinib, lapatinib, nilotinib, topotecan) 
• medicines to reduce the activity of the immune system (e.g. sirolimus) 
• medicines to treat HIV (e.g. maraviroc) 
• medicines to treat and prevent blood clots (e.g. dabigatran etexilate) 
• medicines to treat angina – chest pain caused by the heart (e.g. ranolazine) 
• medicines to reduce blood pressure (e.g. talinolol, aliskiren, ambrisentan) 
• medicines to regulate heart rhythm (e.g. digoxin) 
• medicines to treat diabetes (e.g. saxagliptin, sitagliptin) 
• medicines to treat gout (e.g. colchicine) 
• medicines to treat low blood sodium level (e.g. tolvaptan) 
• anti-histamine medicines to treat hayfever (e.g. fexofenadine) 

  
Lojuxta with food, drink and alcohol 

• Do not drink any type of grapefruit juice. 
• The use of alcohol during Lojuxta treatment is not recommended. 
• Your dose of Lojuxta may need to be adjusted if you consume peppermint oil or Seville 

oranges. 
• To lower the chance of stomach problems, you must stay on a low-fat diet whilst taking this 

medicine. Work with a dietitian to learn what you can eat while taking Lojuxta. 
  



47 

Pregnancy and breast-feeding 
Do not take this medicine if you are pregnant, trying to get pregnant, or think you may be pregnant, as 
there is a possibility that it could harm an unborn baby. If you get pregnant while taking this medicine, 
call your doctor immediately and stop taking the capsules. 
 
Pregnancy 

• Before starting treatment you should confirm you are not pregnant and are using effective 
contraception, as advised by your doctor. If you use contraceptive pills and suffer from an 
episode of diarrhoea or vomiting that lasts more than 2 days, you must use an alternative 
method of contraception (e.g. condoms, diaphragm) for 7 days following resolution of 
symptoms. 

• If, after you have started treatment with Lojuxta, you decide that you would like to become 
pregnant, please inform your doctor, as your treatment will need to be changed. 

 
Breast-feeding 

• It is not known if Lojuxta is passed into breast milk. Please tell your doctor if you are breast-
feeding or planning to breast-feed. Your doctor may advise you to stop taking Lojuxta or to 
stop breast-feeding. 

 
Driving and using machines 
Your treatment may affect your ability to drive or use machines. If you feel dizzy during treatment 
then do not drive or use machines until you feel better. 
 
Lojuxta contains lactose 
If you have been told by your doctor that you have an intolerance to some sugars, contact your doctor 
before taking this medicine. 
 
 
3. How to take Lojuxta 
 
Always take this medicine exactly as your doctor or pharmacist has told you. Check with your doctor 
or pharmacist if you are not sure. These capsules should be given to you by a doctor experienced in 
the treatment of lipid disorders who will also monitor you regularly. 
 
The recommended starting dose is a 5 mg capsule each day. Your doctor may increase your dose 
slowly over time, up to a maximum of 60 mg each day. Your doctor will tell you: 

• what dose to take and for how long. 
• when to increase or decrease your dose. 

Do not change the dose yourself. 
 

• Take this medicine once a day at bedtime with a glass of water at least 2 hours after your 
evening meal (see section 2 under ‘Lojuxta with food, drink and alcohol’).  

• Do not take this medicine with food, as taking these capsules with food can cause stomach 
problems.  

• If you take another medicine that lowers cholesterol by binding bile acids, such as colesevelam 
or cholestyramine, take the medicine that binds bile acids at least 4 hours before or 4 hours 
after you take Lojuxta.  

 
Because of the possibility of interactions with other medicines, your doctor may change the time of 
day you take your medicines. Alternatively, your doctor may decrease your dose of Lojuxta. Inform 
your doctor of any change in the medicines you are taking. 
 
You also need to take daily vitamin E and essential fatty acid (omega-3 and omega-6) supplements 
while taking this medicine. The usual dose that you will need to take is listed below. Ask your doctor, 
or dietitian how to obtain these supplements. See section 2 under ‘ Lojuxta with food, drink and 
alcohol’. 
 



48 

Daily Amount  
Vitamin E  400 IU* 
Omega-3  Approximately  
EPA  110 mg* 
DHA 80 mg 
ALA 210 mg 
Omega-6   
Linoleic acid 200 mg  
* IU – international units, mg - milligrams 

 
If you take more Lojuxta than you should  
Contact your doctor or pharmacist immediately.  
 
If you forget to take Lojuxta 
Just take your normal dose at the usual time the next day. Do not take a double dose to make up for a 
forgotten dose.  
 
If you stop taking Lojuxta 
If you stop taking this medicine your cholesterol may rise again. You should contact your doctor 
before you stop taking this medicine. 
 
If you have any further questions on the use of this medicine, ask your doctor or pharmacist. 
 
 
4. Possible side effects 
 
Like all medicines, this medicine can cause side effects, although not everybody gets them. 
 
Serious side effects: 

• abnormal blood tests for liver function have been reported commonly (may affect up to 1 in 
10 people). The signs and symptoms of liver problems include: 

o nausea (feeling sick) 
o vomiting (being sick) 
o stomach pain 
o muscle aches and pains 
o fever 
o skin or whites of your eyes turn yellow 
o being more tired than usual 
o feeling like you have the flu 

 
Tell your doctor immediately if you have any of these symptoms as your doctor may decide to stop 
the treatment. 
 
The following other side effects have also occurred: 
 
Very common (may affect more than 1 in 10 people):  

• diarrhoea 
• nausea and vomiting (feeling or being sick) 
• stomach pain, discomfort or stomach bloating 
• decreased appetite 
• indigestion 
• flatulence (wind) 
• constipation  
• weight loss 

 



49 

Common (may affect up to 1 in 10 people):  
• inflammation of the stomach and intestine that causes diarrhoea and vomiting  
• regurgitation (bringing food back up) 
• burping 
• feeling of incomplete defaecation (bowel movement), urgent need to defaecate 
• bleeding from your rectum (back passage) or blood in your stool  
• dizziness, headache, migraine 
• tiredness, lack of energy or general weakness 
• enlarged, damaged or fatty liver 
• purple discoloration of the skin, solid bumps on the skin, rash, yellow bumps on the skin 
• changes to blood clotting tests 
• changes to blood cell count  
• decrease in levels of potassium, carotene, vitamin E, vitamin K in your blood 
• muscle spasms 
 

Uncommon (may affect up to 1 in 100 people):  
• flu or cold, fever, inflammation of your sinuses, cough 
• low red blood cell count (anaemia) 
• dehydration, dry mouth 
• increased appetite 
• burning or prickling of the skin 
• swelling of the eye 
• ulcer or sore spot in the throat 
• vomiting blood 
• dry skin 
• blister 
• excessive sweating 
• joint pain or swelling, pain in hands or feet 
• muscle pain 
• blood or protein in the urine 
• chest pain 
• changes to your walking (gait)  
• abnormal lung function test 

 
Not known (frequency cannot be estimated from the available data) 

• hair loss (alopecia) 
• muscle pain (myalgia) 
• loss of fluid that may cause headache, dry mouth, dizziness, tiredness or unconsciousness 

(dehydration) 
 
Reporting of side effects 
If you get any side effects, talk to your doctor or pharmacist. This includes any possible side effects 
not listed in this leaflet. You can also report side effects directly via the national reporting system 
listed in Appendix V. By reporting side effects you can help provide more information on the safety of 
this medicine. 
 
 
5. How to store Lojuxta 
 
Keep this medicine out of the sight and reach of children. 
 
Do not use this medicine after the expiry date which is stated on the label or carton after “EXP”. The 
expiry date refers to the last day of that month. 
 

http://www.ema.europa.eu/docs/en_GB/document_library/Template_or_form/2013/03/WC500139752.doc


50 

Store below 30°C.  
Keep the bottle tightly closed in order to protect from moisture. 
 
Do not throw away any medicines via wastewater or household waste. Ask your pharmacist how to 
throw away medicines you no longer use. These measures will help to protect the environment. 
 
 
6. Contents of the pack and other information 
 
What Lojuxta contains  

• The active substance is lomitapide.  
Lojuxta 30 mg: each hard capsule contains lomitapide mesylate equivalent to 30 mg lomitapide. 
Lojuxta 40 mg: each hard capsule contains lomitapide mesylate equivalent to 40 mg lomitapide. 
Lojuxta 60 mg: each hard capsule contains lomitapide mesylate equivalent to 60 mg lomitapide. 

• The other ingredients are: pregelatinized starch, sodium starch glycolate (Type A), 
microcrystalline cellulose, lactose monohydrate, silica colloidal anhydrous and magnesium 
stearate (for information on lactose monohydrate, see section 2 under ‘Lojuxta contains 
lactose’).  

 
Capsule shell: 
• The capsule shell for the 30 mg capsule contains gelatin, titanium dioxide (E171), red iron oxide 

(E172) and yellow iron oxide (E172).  
• The capsule shell for the 40 mg capsule contains gelatin, titanium dioxide (E171) and yellow 

iron oxide (E172).  
• The capsule shell for the 60 mg capsule contains gelatin, titanium dioxide (E171) and yellow 

iron oxide (E172). 
• All capsules have edible black ink for printing. 

 
What Lojuxta looks like and contents of the pack 

• Lojuxta 30 mg is an orange cap/yellow body hard capsule with “30 mg” printed on the body and 
“A733” printed on the cap in black ink. 

• Lojuxta 40 mg is a yellow cap/white body hard capsule with “40 mg” printed on the body and 
“A733” printed on the cap in black ink. 

• Lojuxta 60 mg is a yellow cap/yellow body hard capsule with “60 mg” printed on the body and 
“A733” printed on the cap in black ink. 
 

Pack sizes are:  
28 capsules  
 
Marketing Authorisation Holder 
Amryt Pharmaceuticals DAC 
90 Harcourt Street 
Dublin 2 
Ireland 
 
Manufacturer 
HÄLSA Pharma GmbH 
Hafenweg 18-20 
48155 Münster 
Germany 
 



51 

For any information about this medicine, please contact the local representative of the Marketing 
Authorisation Holder: 
 
België/Belgique/Belgien 
Amryt Pharmaceuticals DAC 
Tél/Tel: +800 44 474447  
Tél/Tel: +44 1604 549952 
medinfo@amrytpharma.com 
 

Lietuva 
Amryt Pharmaceuticals DAC 
Tel: +800 44 474447 
Tel: +44 1604 549952 
medinfo@amrytpharma.com 

България 
Amryt Pharmaceuticals DAC 
Teл.: +44 1604 549952 
medinfo@amrytpharma.com 
 

Luxembourg/Luxemburg 
Amryt Pharmaceuticals DAC 
Tel: +800 44 474447 
Tel: +44 1604 549952 
medinfo@amrytpharma.com 

 
Česká republika 
Amryt Pharmaceuticals DAC 
Tel: +800 44 474447  
Tel: +44 1604 549952 
medinfo@amrytpharma.com 
 

 
Magyarország 
Amryt Pharmaceuticals DAC 
Tel: +800 44 474447  
Tel: +44 1604 549952 
medinfo@amrytpharma.com 

Danmark 
Amryt Pharmaceuticals DAC 
Tlf: +800 44 474447 
Tlf: +44 1604 549952 
medinfo@amrytpharma.com 
 

Malta 
Amryt Pharmaceuticals DAC 
Tel: +44 1604 549952 
medinfo@amrytpharma.com 

Deutschland 
Amryt Pharmaceuticals DAC 
Tel: +800 44 474447  
Tel: +44 1604 549952 
medinfo@amrytpharma.com 
 

Nederland 
Amryt Pharmaceuticals DAC 
Tel: +800 44 474447  
Tel: +44 1604 549952 
medinfo@amrytpharma.com 

Eesti 
Amryt Pharmaceuticals DAC 
Tel: +800 44 474447  
Tel: +44 1604 549952 
medinfo@amrytpharma.com 
 

Norge 
Amryt Pharmaceuticals DAC 
Tlf: +44 1604 549952 
medinfo@amrytpharma.com 

Ελλάδα 
Amryt Pharmaceuticals DAC 
Tηλ: +800 44 474447  
Tηλ: +44 1604 549952 
medinfo@amrytpharma.com 
 

Österreich 
Amryt Pharmaceuticals DAC 
Tel: +800 44 474447 
Tel: +44 1604 549952 
medinfo@amrytpharma.com 
 

España 
Amryt Pharmaceuticals DAC 
Tel: +800 44 474447 
Tel: +44 1604 549952 
medinfo@amrytpharma.com 
  

Polska 
Amryt Pharmaceuticals DAC 
Tel: +800 44 474447  
Tel: +44 1604 549952 
medinfo@amrytpharma.com 

France 
Amryt Pharmaceuticals DAC 
Tél: +800 44 474447  
Tel: +44 1604 549952 
medinfo@amrytpharma.com 
 

Portugal 
Amryt Pharmaceuticals DAC 
Tel: +800 44 474447 
Tel: +44 1604 549952 
medinfo@amrytpharma.com  
 

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52 

Hrvatska 
Amryt Pharmaceuticals DAC 
Tel: +44 1604 549952 
medinfo@amrytpharma.com 
 

România 
Amryt Pharmaceuticals DAC 
Tel: +44 1604 549952 
medinfo@amrytpharma.com 
 

Ireland 
Amryt Pharmaceuticals DAC 
Tel: +800 44 474447  
Tel: +44 1604 549952 
medinfo@amrytpharma.com 
 

Slovenija 
Amryt Pharmaceuticals DAC 
Tel: +44 1604 549952 
medinfo@amrytpharma.com 

Ísland 
Amryt Pharmaceuticals DAC 
Sími: +44 1604 549952 
medinfo@amrytpharma.com 
 

Slovenská republika 
Amryt Pharmaceuticals DAC 
Tel: +44 1604 549952 
medinfo@amrytpharma.com 

Italia 
Amryt Pharma Italy SRL 
Tel: +800 44 474447 
Tel: +44 1604 549952 
medinfo@amrytpharma.com 
 

Suomi/Finland 
Amryt Pharmaceuticals DAC 
Tel: +800 44 474447   
Tel: +44 1604 549952 
medinfo@amrytpharma.com 

Κύπρος 
Amryt Pharmaceuticals DAC 
Tηλ: +800 44 474447  
Tηλ: +44 1604 549952 
medinfo@amrytpharma.com 
 

Sverige 
Amryt Pharmaceuticals DAC 
Tel: +44 1604 549952 
medinfo@amrytpharma.com 
 

Latvija 
Amryt Pharmaceuticals DAC 
Tel: +44 1604 549952 
medinfo@amrytpharma.com 
 

United Kingdom 
Amryt Pharmaceuticals DAC 
Tel: +800 44 474447 (freephone) 
Tel: +44 1604 549952 
medinfo@amrytpharma.com 

 
 
This leaflet was last revised in  
 
Other sources of information 
This medicine has been authorised under ‘exceptional circumstances’. This means that because of the 
rarity of this disease it has been impossible to get complete information on this medicine. The 
European Medicines Agency will review any new information on this medicine every year and this 
leaflet will be updated as necessary. 
 
Detailed information on this medicine is available on the European Medicines Agency web site: 
http://www.ema.europa.eu.  
 
 
 
 

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	SUMMARY OF PRODUCT CHARACTERISTICS
	A. MANUFACTURER RESPONSIBLE FOR BATCH RELEASE
	B. CONDITIONS OR RESTRICTIONS REGARDING SUPPLY AND USE
	C. OTHER CONDITIONS AND REQUIREMENTS OF THE MARKETING AUTHORISATION 
	D. CONDITIONS OR RESTRICTIONS WITH REGARD TO THE SAFE AND EFFECTIVE USE OF THE MEDICINAL PRODUCT
	E. SPECIFIC OBLIGATION TO COMPLETE POST-AUTHORISATION MEASURES FOR THE MARKETING AUTHORISATION UNDER EXCEPTIONAL CIRCUMSTANCES 
	A. LABELLING
	B. PACKAGE LEAFLET