Deferiprone Lipomed

1 

 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 

ANNEX I 
 

SUMMARY OF PRODUCT CHARACTERISTICS 



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1. NAME OF THE MEDICINAL PRODUCT 
 
Deferiprone Lipomed 500 mg film-coated tablets 
 
 
2. QUALITATIVE AND QUANTITATIVE COMPOSITION 
 
Each film-coated tablet contains 500 mg deferiprone. 
 
For the full list of excipients, see section 6.1. 
 
 
3. PHARMACEUTICAL FORM 
 
Film-coated tablet. 
 
White to off-white, glossy surface, oval film-coated tablet. The tablet is scored. The tablet can be 
divided into equal doses. 
 
 
4. CLINICAL PARTICULARS 
 
4.1 Therapeutic indications 
 
Deferiprone Lipomed monotherapy is indicated for the treatment of iron overload in patients with 
thalassaemia major when current chelation therapy is contraindicated or inadequate. 
 
Deferiprone Lipomed in combination with another chelator (see section 4.4) is indicated in patients 
with thalassaemia major when monotherapy with any iron chelator is ineffective, or when prevention 
or treatment of life-threatening consequences of iron overload justifies rapid or intensive correction 
(see section 4.2). 
 
4.2 Posology and method of administration 
 
Deferiprone therapy should be initiated and maintained by a physician experienced in the treatment of 
patients with thalassaemia. 
 
Posology 
Deferiprone is usually given as 25 mg/kg body weight, orally, three times a day for a total daily dose 
of 75 mg/kg body weight. Dose per kilogram body weight should be calculated to the nearest half 
tablet. See tables below for recommended doses for body weights at 10 kg increments. 
 
To obtain a dose of about 75 mg/kg/day, use the number of tablets suggested in the following tables 
for the body weight of the patient. Sample body weights at 10 kg increments are listed. 
 
Dose table for Deferiprone Lipomed 500 mg film-coated tablets 

Body weight 
(kg) 

Total daily dose 
(mg) 

Dose 
(mg, three times/day) 

Number of tablets 
(three times/day) 

20 1500 500 1.0 
30 2250 750 1.5 
40 3000 1000 2.0 
50 3750 1250 2.5 
60 4500 1500 3.0 
70 5250 1750 3.5 
80 6000 2000 4.0 
90 6750 2250 4.5 

 



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A total daily dose above 100 mg/kg body weight is not recommended because of the potentially 
increased risk of adverse reactions (see sections 4.4, 4.8, and 4.9). 
 
Dose adjustment 
The effect of Deferiprone Lipomed in decreasing the body iron is directly influenced by the dose and 
the degree of iron overload. After starting Deferiprone Lipomed therapy, it is recommended that serum 
ferritin concentrations, or other indicators of body iron load, be monitored every two to three months 
to assess the long-term effectiveness of the chelation regimen in controlling the body iron load. Dose 
adjustments should be tailored to the individual patient’s response and therapeutic goals (maintenance 
or reduction of body iron burden). Interruption of therapy with deferiprone should be considered if 
serum ferritin falls below 500 µg/l. 
 
Dose adjustments when used with other iron chelators 
In patients for whom monotherapy is inadequate, Deferiprone Lipomed may be used with 
deferoxamine at the standard dose (75 mg/kg/day) but should not exceed 100 mg/kg/day. The product 
information of deferoxamine should be consulted. 
 
Concurrent use of iron chelators is not recommended in patients whose serum ferritin falls below 
500 µg/l due to the risk of excessive iron removal. 
 
Paediatric population 
There are limited data available on the use of deferiprone in children between 6 and 10 years of age, 
and no data on deferiprone use in children under 6 years of age. 
 
Renal impairment 
Dose adjustment is not required in patients with mild, moderate, or severe renal impairment (see 
section 5.2). The safety and pharmacokinetics of Deferiprone Lipomed in patients with end stage renal 
disease are unknown. 
 
Hepatic impairment 
Dose adjustment is not required in patients with mildly or moderately impaired hepatic function (see 
section 5.2). The safety and pharmacokinetics of Deferiprone Lipomed in patients with severe hepatic 
impairment are unknown. 
 
Method of administration  
For oral use. 
 
4.3 Contraindications 
 

• Hypersensitivity to the active substance or to any of the excipients listed in section 6.1. 
• History of recurrent episodes of neutropenia. 
• History of agranulocytosis. 
• Pregnancy (see section 4.6). 
• Breast-feeding (see section 4.6). 
• Due to the unknown mechanism of deferiprone-induced neutropenia, patients must not take 

medicinal products known to be associated with neutropenia or those that can cause 
agranulocytosis (see section 4.5). 

 



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4.4 Special warnings and precautions for use 
 
Neutropenia/Agranulocytosis 
Deferiprone has been shown to cause neutropenia, including agranulocytosis (see section 4.8 
‘Description of selected adverse reactions’). The patient’s absolute neutrophil count (ANC) 
should be monitored every week during the first year of therapy. For patients whose deferiprone 
has not been interrupted during the first year of therapy due to any decrease in the neutrophil 
count, the frequency of ANC monitoring may be extended to the patient’s blood transfusion 
interval (every 2 – 4 weeks) after one year of deferiprone therapy. 
 
The change from weekly ANC monitoring to at the time of transfusion visits after 12 months of 
deferiprone therapy should be considered on an individual patient basis, according to the physician’s 
assessment of the patient’s understanding of the risk minimization measures required during therapy 
(see section 4.4 below). 
 
In clinical trials, weekly monitoring of the neutrophil count has been effective in identifying cases of 
neutropenia and agranulocytosis. Agranulocytosis and neutropenia usually resolve upon 
discontinuation of deferiprone, but fatal cases of agranulocytosis have been reported. If the patient 
develops an infection while on deferiprone, therapy should be immediately interrupted, and an ANC 
obtained without delay. The neutrophil count should then be monitored more frequently. 
 
Patients should be aware to contact their physician if they experience any symptoms indicative 
of infection (such as fever, sore throat and flu-like symptoms). Immediately interrupt 
deferiprone if the patient experiences infection. 
 
Suggested management of cases of neutropenia is outlined below. It is recommended that such a 
management protocol be in place prior to initiating any patient on deferiprone treatment. 
 
Treatment with deferiprone should not be initiated if the patient is neutropenic. The risk of 
agranulocytosis and neutropenia is higher if the baseline ANC is less than 1.5 x 109/l. 
 
For neutropenia events (ANC < 1.5 x 109/l and > 0.5 x 109/l): 
Instruct the patient to immediately discontinue deferiprone and all other medicinal products with a 
potential to cause neutropenia. The patient should be advised to limit contact with other individuals in 
order to reduce the risk of infection. Obtain a complete blood cell (CBC) count, with a white blood 
cell (WBC) count, corrected for the presence of nucleated red blood cells, a neutrophil count, and a 
platelet count immediately upon diagnosing the event and then repeat daily. It is recommended that 
following recovery from neutropenia, weekly CBC, WBC, neutrophil and platelet counts continue to 
be obtained for three consecutive weeks, to ensure that the patient recovers fully. Should any evidence 
of infection develop concurrently with the neutropenia, the appropriate cultures and diagnostic 
procedures should be performed and an appropriate therapeutic regimen instituted. 
 
For agranulocytosis (ANC < 0.5 x 109/l): 
Follow the guidelines above and administer appropriate therapy such as granulocyte colony 
stimulating factor, beginning the same day that the event is identified; administer daily until the 
condition resolves. Provide protective isolation and if clinically indicated, admit patient to the hospital. 
 
Limited information is available regarding re-challenge. Therefore, in the event of neutropenia, re-
challenge is not recommended. In the event of agranulocytosis, re-challenge is contraindicated. 
 
Carcinogenicity/mutagenicity 
In view of the genotoxicity results, a carcinogenic potential of deferiprone cannot be excluded (see 
section 5.3). 
 
Plasma Zn2+ concentration 
Monitoring of plasma Zn2+ concentration, and supplementation in case of a deficiency, is 
recommended. 



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HIV positive or other immunocompromised patients 
No data are available on the use of deferiprone in HIV positive or other immunocompromised patients. 
Given that deferiprone can be associated with neutropenia and agranulocytosis, therapy in 
immunocompromised patients should not be initiated unless potential benefits outweigh potential 
risks. 
 
Renal or hepatic impairment and liver fibrosis 
There are no data available on the use of deferiprone in patients with end stage renal disease or severe 
hepatic impairment (see section 5.2). Caution must be exercised in patients with end stage renal 
disease or severe hepatic dysfunction. Renal and hepatic function should be monitored in these patient 
populations during deferiprone therapy. If there is a persistent increase in serum alanine 
aminotransferase (ALT), interruption of deferiprone therapy should be considered. 
 
In thalassaemia patients there is an association between liver fibrosis and iron overload and/or 
hepatitis C. Special care must be taken to ensure that iron chelation in patients with hepatitis C is 
optimal. In these patients careful monitoring of liver histology is recommended. 
 
Discoloration of urine 
Patients should be informed that their urine may show a reddish/brown discoloration due to the 
excretion of the iron-deferiprone complex. 
 
Chronic overdose and neurological disorders 
Neurological disorders have been observed in children treated with more than 2.5 times the maximum 
recommended dose for several years but have also been observed with standard doses of deferiprone. 
Prescribers are reminded that the use of doses above 100 mg/kg/day is not recommended. Deferiprone 
use should be discontinued if neurological disorders are observed (see sections 4.8 and 4.9). 
 
Combined use with other iron chelators 
The use of combination therapy should be considered on a case-by-case basis. The response to therapy 
should be assessed periodically, and the occurrence of adverse events closely monitored. Fatalities and 
life-threatening situations (caused by agranulocytosis) have been reported with deferiprone in 
combination with deferoxamine. Combination therapy with deferoxamine is not recommended when 
monotherapy with either chelator is adequate or when serum ferritin falls below 500 µg/l. Limited data 
are available on the combined use of deferiprone and deferasirox, and caution should be applied when 
considering the use of such combination. 
 
Excipients 
This medicinal product contains less than 1 mmol sodium (23 mg) per tablet and that is to say 
essentially ‘sodium-free’. 
 
4.5 Interaction with other medicinal products and other forms of interaction 
 
Due to the unknown mechanism of deferiprone-induced neutropenia, patients must not take medicinal 
products known to be associated with neutropenia or those that can cause agranulocytosis (see section 
4.3). 
 
Since deferiprone binds to metallic cations, the potential exists for interactions between deferiprone 
and trivalent cation-dependent medicinal products such as aluminium-based antacids. Therefore, it is 
not recommended to concomitantly ingest aluminium-based antacids and deferiprone. 
 
The safety of concurrent use of deferiprone and vitamin C has not been formally studied. Based on the 
reported adverse interaction that can occur between deferoxamine and vitamin C, caution should be 
used when administering deferiprone and vitamin C concurrently. 
 



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4.6 Fertility, pregnancy and lactation 
 
Pregnancy 
There are no adequate data from the use of deferiprone in pregnant women. Studies in animals have 
shown reproductive toxicity (see section 5.3). The potential risk for humans is unknown. 
 
Women of childbearing potential must be advised to avoid pregnancy due to the clastogenic and 
teratogenic properties of the medicinal product. These women should be advised to take contraceptive 
measures and must be advised to immediately stop taking deferiprone if they become pregnant or plan 
to become pregnant (see section 4.3). 
 
Breast-feeding 
It is not known whether deferiprone is excreted in human milk. No prenatal and postnatal reproductive 
studies have been conducted in animals. Deferiprone must not be used by breast-feeding mothers. If 
treatment is unavoidable, breast-feeding must be stopped (see section 4.3). 
 
Fertility 
No effects on fertility or early embryonic development were noted in animals (see section 5.3). 
 
4.7 Effects on ability to drive and use machines 
 
Not relevant. 
 
4.8 Undesirable effects 
 
Summary of the safety profile 
The most common adverse reactions reported during therapy with deferiprone in clinical trials were 
nausea, vomiting, abdominal pain, and chromaturia, which were reported in more than 10% of 
patients. The most serious adverse reaction reported in clinical trials with deferiprone was 
agranulocytosis, defined as an absolute neutrophil count less than 0.5 x 109/l, which occurred in 
approximately 1% of patients. Less severe episodes of neutropenia were reported in approximately 5% 
of patients. 
 



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Tabulated list of adverse reactions 
Adverse reaction frequencies: Very common (≥1/10), Common (≥1/100 to <1/10), not known (cannot 
be estimated from the available data). 
 
SYSTEM ORGAN CLASS VERY COMMON 

(≥1/10) 
COMMON 
(≥1/100 to <1/10) 

FREQUENCY 
NOT KNOWN 

Blood and lymphatic system 
disorders 

 Neutropenia 
Agranulocytosis 

 

Immune system disorders   Hypersensitivity 
reactions 

Metabolism and nutrition 
disorders 

 Increased appetite  

Nervous system disorders  Headache  
Gastrointestinal disorders Nausea 

Abdominal pain 
Vomiting 

Diarrhoea  

Skin and subcutaneous tissue 
disorders 

  Rash 
Urticaria 

Musculoskeletal and 
connective tissue disorders 

 Arthralgia  

Renal and urinary disorders Chromaturia   
General disorders and 
administration site conditions 

 Fatigue  

Investigations  Increased liver 
enzymes 

 

 
Description of selected adverse reactions 
The most serious adverse reaction reported in clinical trials with deferiprone is agranulocytosis 
(neutrophils <0.5 x 109/l), with an incidence of 1.1% (0.6 cases per 100 patient-years of treatment) 
(see section 4.4). Data from pooled clinical studies in patients with systemic iron overload show that 
63% of the episodes of agranulocytosis occurred within the first six months of treatment, 74% within 
the first year and 26% after one year of therapy. The median time to onset of the first episode of 
agranulocytosis was 190 days (ranged 22 days- 17.6 years) and median duration was 10 days in 
clinical trials. A fatal outcome was observed in 8.3% of the reported episodes of agranulocytosis from 
clinical trials and post-marketing experience. 
 
The observed incidence of the less severe form of neutropenia (neutrophils <1.5 x 109/l) is 4.9% (2.5 
cases per 100 patient-years). This rate should be considered in the context of the underlying elevated 
incidence of neutropenia in thalassaemia patients, particularly in those with hypersplenism. 
 
Episodes of diarrhoea, mostly mild and transient, have been reported in patients treated with 
deferiprone. Gastrointestinal effects are more frequent at the beginning of therapy and resolve in most 
patients within a few weeks without the discontinuation of treatment. In some patients it may be 
beneficial to reduce the dose of deferiprone and then scale it back up to the former dose. Arthropathy 
events, which ranged from mild pain in one or more joints to severe arthritis with effusion and 
significant disability, have also been reported in patients treated with deferiprone. Mild arthropathies 
are generally transient. 
 
Increased levels of serum liver enzymes have been reported in some patients taking deferiprone. In the 
majority of these patients, the increase was asymptomatic and transient, and returned to baseline 
without discontinuation or decreasing the dose of deferiprone (see section 4.4). 
 
Some patients experienced progression of fibrosis associated with an increase in iron overload or 
hepatitis C. 
 
Low plasma zinc levels have been associated with deferiprone in a minority of patients. The levels 
normalised with oral zinc supplementation. 



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Neurological disorders (such as cerebellar symptoms, diplopia, lateral nystagmus, psychomotor 
slowdown, hand movements and axial hypotonia) have been observed in children who had been 
voluntarily prescribed more than 2.5 times the maximum recommended dose of 100 mg/kg/day for 
several years. Episodes of hypotonia, instability, inability to walk, and hypertonia with inability of 
limb movement have been reported in children in the post-marketing setting with standard doses of 
deferiprone. The neurological disorders progressively regressed after deferiprone discontinuation (see 
sections 4.4 and 4.9). 
 
The safety profile of combination therapy (deferiprone and deferoxamine) observed in clinical trials, 
post-marketing experience or published literature was consistent with that characterized for 
monotherapy. 
 
Data from the pooled safety database from clinical trials (1343 patient-years exposure to deferiprone 
monotherapy and 244 patient-years exposure to deferiprone and deferoxamine) showed statistically 
significant (p<0.05) differences in the incidence of adverse reactions based on System Organ Class for 
“Cardiac disorders”, “Musculoskeletal and connective tissue disorders” and “Renal and urinary 
disorders”. The incidences of “Musculoskeletal and connective tissue disorders” and “Renal and 
urinary disorders” were lower during combination therapy than monotherapy, whereas the incidence 
of “Cardiac disorders” was higher during combination therapy than monotherapy. The higher rate of 
“Cardiac disorders” reported during combination therapy than monotherapy was possibly due to the 
higher incidence of pre-existing cardiac disorders in patients who received combination therapy. 
Careful monitoring of cardiac events in patients on combination therapy is warranted (see section 4.4). 
 
Paediatric population 
The incidences of adverse reactions experienced by 18 children and 97 adults treated with combination 
therapy were not significantly different between the two age groups except in the incidence of 
arthropathy (11.1% in children vs. none in adults, p=0.02). Evaluation of rate of reactions per 
100 patient-years of exposure showed that only the rate of diarrhoea was significantly higher in 
children (11.1) than in adults (2.0, p=0.01). 
 
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 
 
No cases of acute overdose have been reported. However, neurological disorders (such as cerebellar 
symptoms, diplopia, lateral nystagmus, psychomotor slowdown, hand movements and axial 
hypotonia) have been observed in children who had been voluntarily prescribed more than 2.5 times 
the maximum recommended dose of 100 mg/kg/day for several years. The neurological disorders 
progressively regressed after deferiprone discontinuation. 
 
In case of overdose, close clinical supervision of the patient is required. 
 
 
5. PHARMACOLOGICAL PROPERTIES 
 
5.1  Pharmacodynamic properties 
 
Pharmacotherapeutic group: Iron chelating agents, ATC code: V03AC02 
 
Mechanism of action 
The active substance is deferiprone (3-hydroxy-1,2-dimethylpyridin-4-one), a bidentate ligand which 
binds iron in a 3:1 molar ratio. 

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


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Pharmacodynamic effects 
Clinical studies have demonstrated that deferiprone is effective in promoting iron excretion and that a 
dose of 25 mg/kg three times per day can prevent the progression of iron accumulation as assessed by 
serum ferritin, in patients with transfusion-dependent thalassaemia. Data from the published literature 
on iron balance studies in patients with thalassaemia major show that the use of deferiprone 
concurrently with deferoxamine (co-administration of both chelators during the same day, either 
simultaneously or sequentially, e.g., deferiprone during the day and deferoxamine during the night), 
promotes greater iron excretion than either drug alone. Doses of deferiprone in those studies ranged 
from 50 to 100 mg/kg/day and doses of deferoxamine from 40 to 60 mg/kg/day. However, chelation 
therapy may not necessarily protect against iron-induced organ damage. 
 
Clinical efficacy and safety 
Studies LA16-0102, LA-01 and LA08-9701 compared the efficacy of deferiprone with that of 
deferoxamine in controlling serum ferritin in transfusion-dependent thalassaemia patients. Deferiprone 
and deferoxamine were equivalent in promoting a net stabilization or reduction of body iron load, 
despite the continuous transfusional iron administration in those patients (no difference in proportion 
of patients with a negative trend in serum ferritin between the two treatment groups by regression 
analysis; p>0.05). 
 
Study LA37-1111 was conducted to evaluate the effect of single therapeutic (33 mg/kg) and 
supratherapeutic (50 mg/kg) oral doses of deferiprone on the cardiac QT interval duration in healthy 
subjects. The maximum difference between the LS means of the therapeutic dose and placebo was 
3.01 ms (95% one-sided UCL: 5.01 ms), and between the LS means of the supratherapeutic dose and 
placebo was 5.23 ms (95% one-sided UCL: 7.19 ms). Deferiprone was concluded to produce no 
significant prolongation of the QT interval. 
 
5.2 Pharmacokinetic properties 
 
Absorption 
Deferiprone is rapidly absorbed from the upper part of the gastrointestinal tract. Peak serum 
concentration occurs 45 to 60 minutes following a single dose in fasted patients. This may be extended 
to 2 hours in fed patients. 
 
Following a dose of 25 mg/kg, lower peak serum concentrations have been detected in patients in the 
fed state (85 µmol/l) than in the fasting state (126 µmol/l), although there was no decrease in the 
amount of deferiprone absorbed when it was given with food. 
 
Biotransformation 
Deferiprone is metabolised predominantly to a glucuronide conjugate. This metabolite lacks iron-
binding capability due to inactivation of the 3-hydroxy group of deferiprone. Peak serum 
concentrations of the glucuronide occur 2 to 3 hours after administration of deferiprone. 
 
Elimination 
In humans, deferiprone is eliminated mainly via the kidneys; 75% to 90% of the ingested dose is 
reported as being recovered in the urine in the first 24 hours, in the form of free deferiprone, the 
glucuronide metabolite and the iron-deferiprone complex. A variable amount of elimination via the 
faeces has been reported. The elimination half-life in most patients is 2 to 3 hours. 
 
Renal impairment 
An open-label, non-randomized, parallel group clinical study was conducted to evaluate the effect of 
impaired renal function on the safety, tolerability, and pharmacokinetics of a single 33 mg/kg oral 
dose of deferiprone. Subjects were categorized into 4 groups based on estimated glomerular filtration 
rate (eGFR): healthy volunteers (eGFR ≥ 90 mL/min/1.73m2), mild renal impairment (eGFR 
60-89 mL/min/1.73m2), moderate renal impairment (eGFR 30–59 mL/min/1.73m2), and severe renal 
impairment (eGFR 15–29 mL/min/1.73m2). Systemic exposure to deferiprone and to its metabolite 
deferiprone 3-O-glucuronide was assessed by the PK parameters Cmax and AUC. 



10 

 
Regardless of the degree of renal impairment, the majority of the dose of deferiprone was excreted in 
the urine over the first 24 hours as deferiprone 3-O-glucuronide. No significant effect of renal 
impairment was seen on systemic exposure to deferiprone. Systemic exposure to the inactive 3-O-
glucuronide increased with decreasing eGFR. Based on the results of this study, no adjustment of the 
deferiprone dosage regimen is required in patients with impaired renal function. The safety and 
pharmacokinetics of deferiprone in patients with end stage renal disease is unknown. 
 
Hepatic impairment 
An open-label, non-randomized, parallel group clinical study was conducted to evaluate the effect of 
impaired hepatic function on the safety, tolerability, and pharmacokinetics of a single 33 mg/kg oral 
dose of deferiprone. Subjects were categorized into 3 groups based on the Child-Pugh classification 
score: healthy volunteers, mild hepatic impairment (Class A: 5– 6 points), and moderate hepatic 
impairment (Class B: 7– 9 points). Systemic exposure to deferiprone and to its metabolite deferiprone 
3-O-glucuronide was assessed by the PK parameters Cmax and AUC. Deferiprone AUCs did not differ 
between treatment groups, but Cmax was decreased by 20% in mildly or moderately hepatically 
impaired subjects compared with healthy volunteers. Deferiprone-3-O-glucuronide AUC was 
decreased by 10% and Cmax by 20% in mildly and moderately impaired subjects compared with 
healthy volunteers. A serious adverse event of acute liver and renal injury was seen in one subject with 
moderate hepatic impairment. Based on the results of this study, no adjustment of the deferiprone 
dosage regimen is required in patients with mildly or moderately impaired hepatic function.  
 
The influence of severe hepatic impairment on the pharmacokinetics of deferiprone and deferiprone 
3-O-glucuronide has not been evaluated. The safety and pharmacokinetics of deferiprone in patients 
with severe hepatic impairment is unknown. 
 
5.3 Preclinical safety data 
 
Non-clinical studies have been conducted in animal species including mice, rats, rabbits, dogs and 
monkeys. 
 
The most common findings in non-iron-loaded animals at doses of 100 mg/kg/day and above were 
hematologic effects such as bone marrow hypocellularity, and decreased WBC, RBC and/or platelet 
counts in peripheral blood. 
 
Atrophy of the thymus, lymphoid tissues, and testis, and hypertrophy of the adrenals, were reported at 
doses of 100 mg/kg/day or greater in non-iron-loaded animals. 
 
No carcinogenicity studies in animals have been conducted with deferiprone. The genotoxic potential 
of deferiprone was evaluated in a set of in vitro and in vivo tests. Deferiprone did not show direct 
mutagenic properties; however, it did display clastogenic characteristics in in vitro assays and in 
animals. 
 
Deferiprone was teratogenic and embryotoxic in reproductive studies in non-iron-loaded pregnant rats 
and rabbits at doses at least as low as 25 mg/kg/day. No effects on fertility or early embryonic 
development were noted in non-iron-loaded male and female rats that received deferiprone orally at 
doses of up to 75 mg/kg twice daily for 28 days (males) or 2 weeks (females) prior to mating and until 
termination (males) or through early gestation (females). In females, an effect on the oestrous cycle 
delayed time to confirmed mating at all doses tested. 
 
No prenatal and postnatal reproductive studies have been conducted in animals. 
 
 



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6. PHARMACEUTICAL PARTICULARS 
 
6.1 List of excipients 
 
Tablet core 
Hypromellose 
Croscarmellose sodium 
Silica, colloidal anhydrous 
Microcrystalline cellulose 
Magnesium stearate 
 
Coating 
Hypromellose 
Macrogol 6000 
Titanium dioxide 
 
6.2 Incompatibilities 
 
Not applicable. 
 
6.3 Shelf life 
 
2 years. 
 
6.4 Special precautions for storage 
 
Do not store above 25°C. 
 
6.5 Nature and contents of container 
 
Aluminium/PVC-PVDC blisters in cartons of 100 film-coated tablets. 
 
6.6 Special precautions for disposal 
 
Any unused medicinal product or waste material should be disposed of in accordance with local 
requirements. 
 
 
7. MARKETING AUTHORISATION HOLDER 
 
Lipomed GmbH 
Hegenheimer Strasse 2 
79576 Weil am Rhein 
Germany 
Phone number: +49 7621 1693 472 
Fax number: +49 7621 1693 474 
Electronic mail: lipomed@lipomed.com 
 
 
8. MARKETING AUTHORISATION NUMBER(S)  
 
EU/1/18/1310/001 
 
 
9. DATE OF FIRST AUTHORISATION/RENEWAL OF THE AUTHORISATION 
 
Date of first authorisation: 20 September 2018 



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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/


13 

 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 

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 

 
 



14 

A. MANUFACTURER(S) RESPONSIBLE FOR BATCH RELEASE 
 
Name and address of the manufacturer(s) responsible for batch release 

Lipomed GmbH 
Hegenheimer Strasse 2  
79576 Weil am Rhein 
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 ensure that in each Member State where Deferiprone Lipomed is marketed, all 
patients/carers who are expected to use Deferiprone Lipomed are provided with the Patient/Carer 
reminder card as a part of the outer packaging. 
 
The Patient/Carer reminder card shall contain the following key messages (full text is included in 
Annex IIIA of the marketing authorisation): 
 

• To increase patient awareness of the importance of regular monitoring of the neutrophil 
count during treatment with Deferiprone Lipomed 

• To increase patient awareness of the significance of any symptoms of infection while taking 
Deferiprone Lipomed 

• To warn women of childbearing age to not become pregnant because deferiprone may 
seriously harm the unborn baby 



15 

 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 

ANNEX III 
 

LABELLING AND PACKAGE LEAFLET 



16 

 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 

A. LABELLING 



17 

 
PARTICULARS TO APPEAR ON THE OUTER PACKAGING 
 
OUTER CARTON 
 
 
1. NAME OF THE MEDICINAL PRODUCT 
 
Deferiprone Lipomed 500 mg film-coated tablets 
deferiprone 
 
 
2. STATEMENT OF ACTIVE SUBSTANCE(S) 
 
Each film-coated tablet contains 500 mg deferiprone. 
 
 
3. LIST OF EXCIPIENTS 
 
 
 
 
4. PHARMACEUTICAL FORM AND CONTENTS 
 
100 film-coated tablets 
 
 
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 
 
 
 
 
8. EXPIRY DATE 
 
EXP 
 
 
9. SPECIAL STORAGE CONDITIONS 
 
Do not store above 25°C. 
 



18 

 
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 
 
Lipomed GmbH 
Hegenheimer Strasse 2 
79576 Weil am Rhein 
Germany 
Phone number: +49 7621 1693 472 
Fax number: +49 7621 1693 474 
lipomed@lipomed.com 
 
 
12. MARKETING AUTHORISATION NUMBER(S) 
 
EU/1/18/1310/001 
 
 
 
13. BATCH NUMBER 
 
Lot 
 
 
14. GENERAL CLASSIFICATION FOR SUPPLY 
 
 
 
 
15. INSTRUCTIONS ON USE 
 
 
 
 
16. INFORMATION IN BRAILLE 
 
Deferiprone Lipomed 500 mg 
 
 
17. UNIQUE IDENTIFIER – 2D BARCODE 
 
2D barcode carrying the unique identifier included. 
 
18. UNIQUE IDENTIFIER - HUMAN READABLE DATA 
 
PC:  
SN:  
NN: 



19 

 
MINIMUM PARTICULARS TO APPEAR ON BLISTERS OR STRIPS 
 
BLISTER 
 
 
1. NAME OF THE MEDICINAL PRODUCT 
 
Deferiprone Lipomed 500 mg film-coated tablets 
deferiprone 
 
 
2. NAME OF THE MARKETING AUTHORISATION HOLDER 
 
Lipomed GmbH 
 
 
3. EXPIRY DATE 
 
EXP 
 
 
4. BATCH NUMBER 
 
Lot 
 
 
5. OTHER 
 



20 

 
PATIENT/CARER REMINDER CARD 
 
((Front Cover)) 
 
Important Safety Reminders for Patients 
Taking Deferiprone Lipomed 
 
Prescribing doctor:________________________ 
 
Phone no:_______________________________ 
 

((Back Cover)) 
 
For women of child bearing age 
 
Do not take Deferiprone Lipomed if you are 
pregnant or if you are trying to become pregnant. 
If taken during pregnancy, Deferiprone Lipomed 
may seriously harm the unborn baby. 
 
You must use effective contraception while you 
are taking Deferiprone Lipomed. Ask you doctor 
which method is best for you. If you become 
pregnant while taking Deferiprone Lipomed, stop 
taking the medicine immediately and tell your 
doctor. Do not take Deferiprone Lipomed if you 
are breast-feeding. 

((Inside 1)) 
 
Monitoring your white blood cell count with 
Deferiprone Lipomed 
 
There is a small chance that you may develop 
agranulocytosis (very low white blood cell count) 
while taking Deferiprone Lipomed, which may 
lead to a serious infection. Even though 
agranulocytosis only affects 1 to 2 out of 100 
users, it is important to monitor your blood on a 
regular basis. 

((Inside 2)) 
 
Make sure you do the following: 
 
1. Have your blood monitored on a weekly basis 
for the first one year of treatment with 
deferiprone and as regularly as your doctor 
recommends thereafter. 
 
2. If you get any symptoms of infection such as 
fever, sore throat or flu-like symptoms, 
immediately seek medical attention. Your white 
blood cell count must be checked within 24 hours 
in order to detect potential agranulocytosis. 

 



21 

 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 

B. PACKAGE LEAFLET 



22 

Package leaflet: Information for the patient 
 

Deferiprone Lipomed 500 mg film-coated tablets 
deferiprone 

 
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. 
- Provided in the folding box, you will find a patient/carer reminder card. You should complete 

and read the card carefully and carry it with you. Provide this card to your doctor if you develop 
infection symptoms such as a fever, sore throat or flu-like symptoms. 

 
 
What is in this leaflet 
 
1. What Deferiprone Lipomed is and what it is used for 
2. What you need to know before you take Deferipone Lipomed 
3. How to take Deferiprone Lipomed 
4. Possible side effects  
5. How to store Deferiprone Lipomed 
6. Contents of the pack and other information 
 
 
1. What Deferiprone Lipomed is and what it is used for 
 
Deferiprone Lipomed contains the active substance deferiprone. Deferiprone Lipomed is an iron 
chelator, a type of medicine that removes excess iron from the body. 
 
Deferiprone Lipomed is used to treat iron overload caused by frequent blood transfusions in patients 
with thalassaemia major when current chelation therapy is contraindicated or inadequate. 
 
 
2. What you need to know before you take Deferiprone Lipomed 
 
Do not take Deferiprone Lipomed: 
- if you are allergic to deferiprone or any of the other ingredients of this medicine (listed in 

section 6); 
- if you have a history of repeated episodes of neutropenia (low white blood cell (neutrophil) 

count); 
- if you have a history of agranulocytosis (very low white blood cell (neutrophil) count); 
- if you are currently taking medicines known to cause neutropenia or agranulocytosis (see “Other 

medicines and Deferiprone Lipomed”); 
- if you are pregnant or breast-feeding; 
 



23 

Warnings and precautions  
The most serious side effect that may occur while taking Deferiprone Lipomed is a very low white 
blood cell (neutrophil) count. This condition, known as severe neutropenia or agranulocytosis, has 
occurred in 1 to 2 out of 100 people who have taken deferiprone in clinical studies. Because white 
blood cells help to fight infection, a low neutrophil count may place you at risk of developing a serious 
and potentially life-threatening infection. To monitor for neutropenia, your doctor will ask you to have 
a blood test (to check your white blood cell count) performed regularly, as frequently as every week, 
while you are being treated with Deferiprone Lipomed. It is very important for you to keep all of these 
appointments. Please refer to the patient/carer reminder card provided in the folding box. If you get 
any symptoms of infection such as fever, sore throat or flu-like symptoms, immediately seek medical 
attention. Your white blood cell count must be checked within 24 hours in order to detect potential 
agranulocytosis. 
 
If you are HIV positive or if your liver or kidney function is severely impaired, your doctor may 
recommend additional tests. 
 
Your doctor will also ask you to come in for tests to monitor body iron load. In addition he or she 
might ask you to undergo liver biopsies. 
 
Talk to your doctor or pharmacist before taking Deferiprone Lipomed. 
 
Other medicines and Deferiprone Lipomed 
Do not take medicines known to cause neutropenia or agranulocytosis (see “Do not take Deferiprone 
Lipomed”). Tell your doctor or pharmacist if you are taking, have recently taken, or might take any 
other medicines, including medicines obtained without a prescription. 
 
Do not take aluminium-based antacids at the same time as taking Deferiprone Lipomed. 
 
Please consult with your doctor or pharmacist before taking vitamin C with Deferiprone Lipomed. 
 
Pregnancy and breast-feeding 
Do not take this medicine if you are pregnant or if you are trying to become pregnant. This medicine 
could seriously harm your baby. You must use effective contraception while you are taking 
Deferiprone Lipomed. Ask your doctor which method is best for you. If you become pregnant while 
taking Deferiprone Lipomed, stop taking the medicine immediately and tell your doctor. 
 
Do not use Deferiprone Lipomed if you are breast-feeding. Please refer to the patient/carer reminder 
card provided in the folding box. 
 
Driving and using machines 
Not relevant. 
 
Deferiprone Lipomed contains sodium 
This medicine contains less than 1 mmol sodium (23 mg) per tablet and that is to say essentially 
‘sodium-free’. 
 
 
3. How to take Deferiprone Lipomed 
 
Always take this medicine exactly as your doctor has told you. Check with your doctor or pharmacist 
if you are not sure. The amount of Deferiprone Lipomed that you take will depend on your weight. 
The usual dose is 25 mg/kg, 3 times per day, for a total daily dose of 75 mg/kg. The total daily dose 
should not exceed 100 mg/kg. Take your first dose in the morning. Take your second dose midday. 
Take your third dose in the evening. Deferiprone Lipomed can be taken with or without food; 
however, you may find it easier to remember to take Deferiprone Lipomed if you take it with your 
meals. 
 



24 

If you take more Deferiprone Lipomed than you should 
There are no reports of acute overdose with deferiprone. If you have accidentally taken more than the 
prescribed dose, you should contact your doctor. 
 
If you forget to take Deferiprone Lipomed 
Deferiprone Lipomed will be most effective if you do not miss any doses. If you do miss one dose take 
it as soon as you remember and take your next dose at its regularly scheduled time. If you miss more 
than one dose do not take a double dose to make up for forgotten individual doses, just continue with 
your normal schedule. Do not change your daily dose without first talking to your doctor. 
 
 
4. Possible side effects 
 
Like all medicines, this medicine can cause side effects, although not everybody gets them. 
 
The most serious side effect of Deferiprone Lipomed is a very low white blood cell (neutrophil) count. 
This condition, known as severe neutropenia or agranulocytosis, has occurred in 1 to 2 out of 100 
people who have taken deferiprone in clinical studies. A low white blood cell count can be associated 
with a serious and potentially life-threatening infection. Report immediately to your doctor any 
symptoms of infection such as: fever, sore throat or flu-like symptoms. 
 
Very common side effects (may affect more than 1 in 10 people): 
- abdominal pain 
- nausea 
- vomiting 
- reddish/brown discoloration of urine 
 
If you experience nausea or vomiting, it may help to take your Deferiprone Lipomed with some food. 
Discolored urine is a very common effect and is not harmful. 
 
Common side effects (may affect up to 1 in 10 people): 
- low white blood cell count (agranulocytosis and neutropenia) 
- headache 
- diarrhoea 
- increase in liver enzymes 
- fatigue 
- increase in appetite 
 
Not known (frequency cannot be estimated from the available data): 
- allergic reactions including skin rash or hives 
 
Events of joint pain and swelling ranged from mild pain in one or more joints to severe disability. In 
most cases, the pain disappeared while patients continued taking deferiprone. 
 
Additional side effects in children 
In post-marketing experience with deferiprone, neurological disorders (such as tremors, walking 
disorders, double vision, involuntary muscle contractions, problems with movement coordination) 
have been reported in children who had been voluntarily prescribed more than double the maximum 
recommended dose of 100 mg/kg/day for several years. They recovered from these symptoms after 
discontinuation of deferiprone. 
 
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. 
 

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


25 

 
5. How to store Deferiprone Lipomed 
 
Keep this medicine out of the sight and reach of children. 
 
Do not store above 25°C. 
 
Do not use this medicine after the expiry date which is stated on the carton and on the blister after 
EXP. The expiry date refers to the last day of that month. 
 
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 protect the environment. 
 
 
6. Contents of the pack and other information 
 
What Deferiprone Lipomed contains  
The active substance is deferiprone. Each 500 mg film-coated tablet contains 500 mg deferiprone. 
 
The other ingredients are: 
 
Tablet core: hypromellose, croscarmellose sodium, silica, colloidal anhydrous, microcrystalline 
cellulose, magnesium stearate 
 
Coating: hypromellose, macrogol 6000, titanium dioxide 
 
What Deferiprone Lipomed looks like and contents of the pack 
Deferiprone Lipomed 500 mg film-coated tablets are white to off-white, glossy surface, oval film-
coated tablets. The tablets are scored and breakable in half. Deferiprone Lipomed is packaged in 
blisters. One pack contains 100 film-coated tablets. 
 
Marketing Authorisation Holder and Manufacturer 
Lipomed GmbH 
Hegenheimer Strasse 2 
79576 Weil am Rhein 
Germany 
Phone number: +49 7621 1693 472 
Fax number: +49 7621 1693 474 
Electronic mail: lipomed@lipomed.com 
 
This leaflet was last revised in . 
 
Detailed information on this medicine is available on the European Medicines Agency web site: 
http://www.ema.europa.eu. 
 
 


	SUMMARY OF PRODUCT CHARACTERISTICS
	A. MANUFACTURER(S) 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
	A. LABELLING
	B. PACKAGE LEAFLET