Raxone

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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 
 
Raxone 150 mg film-coated tablets 
 
 
2. QUALITATIVE AND QUANTITATIVE COMPOSITION 
 
Each film-coated tablet contains 150 mg idebenone. 
 
Excipients with known effect 
 
Each film-coated tablet contains 46 mg of lactose (as monohydrate) and 0.23 mg of sunset yellow FCF 
(E110). 
 
For the full list of excipients, see section 6.1. 
 
 
3. PHARMACEUTICAL FORM 
 
Film-coated tablet. 
 
Orange, round, biconvex film-coated tablet of 10 mm diameter, engraved with the Santhera logo on 
one side and ‘150’ on the other side.  
 
 
4. CLINICAL PARTICULARS 
 
4.1 Therapeutic indications 
 
Raxone is indicated for the treatment of visual impairment in adolescent and adult patients with 
Leber’s Hereditary Optic Neuropathy (LHON) (see section 5.1). 
 
4.2 Posology and method of administration 
 
Treatment should be initiated and supervised by a physician with experience in LHON. 
 
Posology 
 
The recommended dose is 900 mg/day idebenone (300 mg, 3 times a day). 
 
No data regarding a continuous treatment with idebenone beyond 6 months is available from 
controlled clinical trials.  
 
Special populations 
 
Elderly 
No specific dose adjustment is required for the treatment of LHON in elderly patients. 
 
Hepatic or renal impairment 
Patients with hepatic or renal impairment have not been investigated. Caution is advised in treatment 
of patients with hepatic or renal impairment (see section 4.4). 
 



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Paediatric population 
The safety and efficacy of Raxone in LHON patients under 12 years of age have not yet been 
established. Currently available data are described in sections 5.1 and 5.2, but no recommendation on 
posology can be made. 
 
Method of administration 
 
Raxone film-coated tablets should be swallowed whole with water. The tablets should not be broken 
or chewed. Raxone should be administered with food because food increases the bioavailability of 
idebenone.  
 
4.3 Contraindications 
 
Hypersensitivity to the active substance or to any of the excipients listed in section 6.1.  
 
4.4 Special warnings and precautions for use 
 
Monitoring 
 
Patients should be regularly monitored according to local clinical practice. 
 
Hepatic or renal impairment 
 
No data are available in these populations. Therefore, caution should be exercised when prescribing 
Raxone to patients with hepatic or renal impairment.  
 
Chromaturia 
 
The metabolites of idebenone are coloured and may cause chromaturia, i.e. a reddish-brown 
discoloration of the urine. This effect is harmless, not associated with haematuria, and does not require 
any adaptation of dose or discontinuation of treatment. Caution should be exercised to ensure that the 
chromaturia does not mask changes of colour due to other reasons (e.g. renal or blood disorders).  
 
Lactose 
 
Raxone contains lactose. Patients with rare hereditary problems of galactose intolerance, total lactase 
deficiency or glucose-galactose malabsorption should not take Raxone. 
 
Sunset yellow 
 
Raxone contains sunset yellow (E110) which may cause allergic reactions. 
 
4.5 Interaction with other medicinal products and other forms of interaction 
 
Data from in vitro studies have demonstrated that idebenone and its metabolite QS10 do not exert 
systemic inhibition of cytochrome P450 isoforms CYP1A2, 2B6, 2C8, 2C9, 2C19, 2D6 and 3A4 at 
clinically relevant concentrations of idebenone or QS10. In addition, no induction of CYP1A2, 
CYP2B6 or CYP3A4 was observed. 
 
In vivo idebenone is a mild inhibitor of CYP3A4. Data from a drug-drug interaction study in 
32 healthy volunteers indicate that on the first day of oral administration of 300 mg idebenone t.i.d., 
the metabolism of midazolam, a CYP3A4 substrate, was not modified when both medicinal products 
were administered together. After repeated administration Cmax and AUC of midazolam were increased 
by 28% and 34%, respectively, when midazolam was administered in combination with 300 mg 
idebenone t.i.d. Therefore, CYP3A4 substrates known to have a narrow therapeutic index such as 
alfentanil, astemizole, terfenadine, cisapride, cyclosporine, fentanyl, pimozide, quinidine, sirolimus, 



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tacrolimus, or ergot alkaloids (ergotamine, dihydroergotamine) should be administered with caution in 
patients receiving idebenone.  
 
Idebenone may inhibit P-glycoprotein (P-gp) with possible exposure increases of, e.g., dabigatran 
etexilate, digoxin or aliskiren. These medicines should be administered with caution in patients 
receiving idebenone.  Idebenone is not a substrate for P-gp in vitro. 
 
4.6 Fertility, pregnancy and lactation 
 
Pregnancy 
 
The safety of idebenone in pregnant women has not been established. Animal studies do not indicate 
direct or indirect harmful effects with respect to reproductive toxicity. Idebenone should only be 
administered to pregnant women or women of child-bearing potential likely to become pregnant if it is 
considered that the benefit of the therapeutic effect outweighs any potential risk.  
 
Breast-feeding 
 
Available pharmacodynamic/toxicological data in animals have shown excretion of idebenone in milk 
(for details see 5.3). A risk to the suckling child cannot be excluded. . A decision must be made 
whether to discontinue breast-feeding or to discontinue/abstain from Raxone therapy taking into 
account the benefit of breast-feeding for the child and the benefit of therapy for the woman. 
 
Fertility 
 
There are no data concerning the effect of exposure to idebenone on human fertility. 
 
4.7 Effects on ability to drive and use machines 
 
Raxone has no or negligible influence on the ability to drive and use machines. 
 
4.8 Undesirable effects  
 
Summary of the safety profile 
 
The most commonly reported adverse reactions to idebenone are mild to moderate diarrhoea (usually 
not requiring the discontinuation of the treatment), nasopharyngitis, cough and back pain.  
 
Tabulated list of adverse reactions 
 
The following adverse reactions emerging from clinical trials in LHON patients or reported post-
marketing in other indications are tabulated below. Frequency groupings are defined to the following 
convention: very common (≥1/10), common (≥1/100 to <1/10), not known (cannot be estimated from 
the available data). 
 

System Organ Class Preferred Term Frequency 
Infections and 
Infestations 

Nasopharyngitis Very common 
Bronchitis Not known 

Blood and lymphatic 
system disorders 

Agranulocytosis, anaemia, leukocytopenia, 
thrombocytopenia, neutropenia 

Not known 
 

Metabolism and nutrition 
disorders 

Blood cholesterol increased, blood 
triglycerides increased 

Not known 
 

Nervous system disorders Seizure, delirium, hallucinations, agitation, 
dyskinesia, hyperkinesia, poriomania, 
dizziness, headache, restlessness, stupor 

Not known 
 

Respiratory, thoracic and 
mediastinal disorders 

Cough Very common  



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Gastrointestinal disorders Diarrhoea Common 
Nausea, vomiting, anorexia, dyspepsia Not known 

Hepatobiliary disorders Alanine aminotransferase increased, 
aspartate aminotransferase increased, blood 
alkaline phosphatase increased, blood 
lactate dehydrogenase increased, gamma- 
lutamyltransferase increased, blood 
bilirubin increased, hepatitis 

Not known 
 

Skin and subcutaneous 
tissue disorders 

Rash, pruritus Not known 
 

Musculoskeletal and 
connective tissue 
disorders 

Back pain Common  
Pain in extremity Not known 

Renal and urinary 
disorders 

Azotaemia, chromaturia Not known 

General disorders and 
administration site 
conditions 

Malaise Not known 

 
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 report of overdose has been received from the RHODOS study. Doses up to 2,250 mg/day have 
been administered in clinical studies showing a safety profile consistent with that reported in 
section 4.8. 
 
There is no specific antidote for idebenone. When needed, supportive symptomatic treatment should 
be given. 
 
 
5. PHARMACOLOGICAL PROPERTIES 
 
5.1  Pharmacodynamic properties 
 
Pharmacotherapeutic group: Psychoanaleptics, Other psychostimulants and nootropics;  
ATC code: N06BX13 
 
Mechanism of action 
 
Idebenone, a short-chain benzoquinone, is an anti-oxidant assumed to be capable of transferring 
electrons directly to complex III of the mitochondrial electron transport chain, thereby circumventing 
complex I and restoring cellular energy (ATP) generation under experimental conditions of complex I 
deficiency. Similarly, in LHON idebenone can transfer electrons directly to complex III of the electron 
transport chain, thereby bypassing complex I which is affected by all three primary mtDNA mutations 
causing LHON, and restoring cellular ATP generation. 
 
According to this biochemical mode of action, idebenone may re-activate viable-but-inactive retinal 
ganglion cells (RGCs) in LHON patients. Depending on the time since symptom onset and the 
proportion of RGCs already affected, idebenone can promote recovery of vision in patients who 
experience vision loss. 
 



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Clinical efficacy and safety 
 
Clinical safety and efficacy of idebenone in LHON have been assessed in one double-blind, 
randomised, placebo-controlled study (RHODOS).  
 
In RHODOS a total of 85 LHON patients, 14-66 years of age, with any of the 3 primary mtDNA 
mutations (G11778A, G3460A or T14484C) and disease duration of not more than 5 years were 
enrolled. Patients received either 900 mg/day Raxone or placebo for a period of 24 weeks (6 months). 
Raxone was given as 3  doses of 300 mg daily, each with meals. 
 
The primary endpoint “best recovery of visual acuity (VA)” was defined as the result from the eye 
experiencing the most positive improvement in VA from baseline to week 24 using ETDRS charts. 
The main secondary endpoint “change in best VA” was measured as the difference between best VA 
in either the left or right eye at 24 weeks compared to baseline (Table 1).  
 
Table 1: RHODOS: Best recovery of VA and change in best VA from baseline to week 24 

Endpoint (ITT) Raxone (N=53) Placebo (N=29) 
Primary endpoint: 
Best recovery of VA  
(mean ± SE; 95%CI) 

logMAR* –0.135 ± 0.041 logMAR –0.071 ± 0.053 
logMAR –0.064, 3 letters (–0.184; 0.055) 

p=0.291 
Main secondary endpoint: 
Change in best VA 
(mean ± SE; 95% CI) 

logMAR –0.035 ± 0.046 logMAR 0.085 ± 0.060 
logMAR –0.120, 6 letters (–0.255; 0.014) 

p=0.078 
Analysis according to Mixed Model of Repeated Measures  
One patient in the placebo group presented with ongoing spontaneous recovery of vision at baseline. Exclusion of this patient 
yielded similar results as in the ITT population; as could be expected, the difference between idebenone and placebo arm was 
slightly larger. 
*logMAR - Logarithm of the Minimum Angle of Resolution 
 
A pre-specified analysis in RHODOS determined the proportion of patients with an eye with baseline 
VA of ≤0.5 logMAR in whom the VA deteriorated to ≥1.0 logMAR. In this small subgroup of patients 
(n=8), 0 of 6 patients in the idebenone group deteriorated to ≥1.0 logMAR whereas 2 of 2 patients in 
the placebo group showed such a deterioration. 
 
In a single-visit observational follow-up study of RHODOS VA assessments from 58 patients obtained 
on average 131 weeks after discontinuation of treatment indicates that the effect of Raxone may be 
maintained.  
 
A post-hoc responder analysis was performed in RHODOS evaluating the proportion of patients who 
had a clinically relevant recovery of VA from baseline in at least one eye, defined as either: (i) 
improvement in VA from unable to read a single letter to able to read at least 5 letters on the ETDRS 
chart; or (ii) improvement in VA by at least 10 letters on the ETDRS chart. Results are shown in 
Table 2 including supporting data from 62 LHON patients using Raxone in an Expanded Access 
Programme (EAP) and from 94 untreated patients in a Case Record Survey (CRS). 
 
Table 2: Proportion of patients with clinically relevant recovery of VA after 6 months from 
baseline  

RHODOS (ITT) RHODOS Raxone (N=53) RHODOS Placebo (N=29) 

Responders (N, %) 16 (30.2 %) 3 (10.3 %) 

EAP and CRS EAP-Raxone (N=62)  CRS-untreated (N=94) 

Responders (N, %) 19 (30.6 %) 18 (19.1 %) 
 
In the EAP the number of responders increased with longer treatment duration, from 19 out of 62 
patients (30.6%) at 6 months to 17 out of 47 patients (36.2%) at 12 months.  
 



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Paediatric population 
 
In clinical trials in Friedreich’s Ataxia, 32 patients between the ages of 8 and 11 years and 91 patients 
between the ages of 12 and 17 years received idebenone at ≥ 900 mg/day for up to 42 months.  
In RHODOS and the EAP in LHON, a total of 3 patients between the ages of 9 and 11 years and 27 
patients between the ages of 12 and 17 years received idebenone at 900 mg/day for up to 33 months. 
 
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. 
 
5.2 Pharmacokinetic properties 
 
Absorption 
 
Food increases the bioavailability of idebenone by approximately 5-7-fold and therefore, Raxone 
should always be administered with food. The tablets should not be broken or chewed.  
 
After oral administration of Raxone, idebenone is rapidly absorbed. On repeat dosing, maximum 
plasma concentrations of idebenone are reached on average within 1 hour (median 0.67 h range: 
0.33-2.00 h).  
 
Distribution 
 
Experimental data have shown that idebenone passes the blood-brain barrier and is distributed at 
significant concentrations in cerebral tissue. Following oral administration pharmacologically relevant 
concentrations of idebenone are detectable in the aqueous humor of the eye. 
 
Biotransformation 
 
Metabolism occurs by means of oxidative shortening of the side chain and by reduction of the quinone 
ring and conjugation to glucuronides and sulphates. Idebenone shows a high first pass metabolism 
resulting in conjugates of idebenone (glucuronides and sulphates (IDE-C)) and the Phase I metabolites 
QS10, QS6, and QS4 as well as their corresponding Phase II metabolites (glucuronides and sulphates 
(QS10+QS10-C, QS6+QS6-C, QS4+QS4-C)). The main metabolites in plasma are IDE-C and 
QS4+QS4-C.  
 
Elimination 
 
Due to the high first-pass effect, the plasma concentrations of idebenone were generally only 
measurable up to 6 hours after oral administration of 750 mg Raxone, given either as a single oral dose 
or after repeated (14 days) t.i.d dosing. The main route of elimination is metabolism, with the majority 
of dose excreted via the kidneys as metabolites. After a single or repeated oral dose of 750 mg 
Raxone, QS4+QS4-C were the most prominent idebenone-derived metabolites in urine, representing 
on average between 49.3% and 68.3% of the total administered dose. QS6+QS6 represented 6.45% to 
9.46%, whereas QS10+QS10-C and IDE+IDE-C were close to 1% or below. 
 



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Linearity/non-linearity 
 
In phase I pharmacokinetic studies, proportional increases in plasma concentrations of idebenone were 
observed for doses from 150 mg to 1050 mg. Neither idebenone nor its metabolites showed time-
dependent pharmacokinetics. 
 
Hepatic or renal impairment 
 
No data are available in these populations.  
 
Paediatric population 
 
Whilst clinical trials experience in paediatrics with LHON is limited to patients of 14 years of age and 
above, pharmacokinetic data from population pharmacokinetic studies, which included paediatric 
Friedreich’s Ataxia patients of age 8 years and above, did not reveal any significant differences in the 
pharmacokinetics of idebenone. 
 
5.3 Preclinical safety data  
 
Non-clinical data reveal no special hazard for humans based on conventional studies of safety 
pharmacology, repeated dose toxicity, genotoxicity, carcinogenic potential, toxicity to reproduction 
and development. 
 
 
6. PHARMACEUTICAL PARTICULARS 
 
6.1 List of excipients 
 
Tablet core 
 
Lactose monohydrate 
Cellulose, microcrystalline 
Croscarmellose sodium 
Povidone (K25) 
Magnesium stearate 
Silica, colloidal anhydrous  
 
Film-coating 
 
Macrogol (3350) 
Poly(vinyl alcohol) 
Talc 
Titanium dioxide  
Sunset yellow FCF (E110) 
 
6.2 Incompatibilities 
 
Not applicable. 
 
6.3 Shelf life 
 
5 years. 
 
6.4 Special precautions for storage 
 
This medicinal product does not require any special storage conditions. 
 



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6.5 Nature and contents of container 
 
White high-density polyethylene bottles with white polypropylene child-resistant tamper-evident 
twist-off caps containing 180 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 
 
Santhera Pharmaceuticals (Deutschland) GmbH  
Marie-Curie Strasse 8 
79539 Lörrach  
Germany  
Tel: +49 (0) 7621 1690 200 
Fax: +49 (0) 7621 1690 201 
Email: office@santhera.com 
 
 
8. MARKETING AUTHORISATION NUMBER(S)  
 
EU/1/15/1020/001 
 
 
9. DATE OF FIRST AUTHORISATION/RENEWAL OF THE AUTHORISATION 
 
Date of first authorisation: 8 September 2015 
Date of latest renewal: 
 
 
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 
 

  



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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 



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A. MANUFACTURER RESPONSIBLE FOR BATCH RELEASE 
 
Name and address of the manufacturer responsible for batch release 
 
Santhera Pharmaceuticals (Deutschland) GmbH 
Marie-Curie Strasse 8 
79539 Lorrach 
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 (PSURs)  
 
The requirements for submission of PSURs 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 marketing authorisation holder (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.  
 
 

 
E. SPECIFIC OBLIGATION TO COMPLETE POST-AUTHORISATION MEASURES 

FOR THE MARKETING AUTHORISATION UNDER EXCEPTIONAL 
CIRCUMSTANCES  

 
This being a marketing authorisation under exceptional circumstances and pursuant to Article 14(8) 
of Regulation (EC) No 726/2004, the MAH shall complete, within the stated timeframe, the 
following measures: 
 
 
 



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Description Due date 
In order to further investigate the benefits of Raxone in the treatment of LHON 
patients, the MAH should conduct and submit the results of an external natural 
history controlled, open-label intervention study to assess the efficacy and safety of 
Raxone in the treatment of LHON patients, including long-term treatment. 

Interim reports should 
be provided at the time 
of annual re-
assessments. 
Due date of final 
report: 31 August 2021  

Non-interventional post-authorisation safety study (PASS): In order to further 
investigate the safety of Raxone in the treatment of LHON patients, the MAH 
should generate data based on an agreed protocol from a drug exposure registry of 
patients prescribed Raxone for the treatment of LHON in clinical practice. The 
registry should also be used to generate data on long-term effectiveness. 

Reports to be provided 
at the time of annual 
re-assessment 

 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 

  



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ANNEX III 
 

LABELLING AND PACKAGE LEAFLET 
  



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A. LABELLING 
  



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PARTICULARS TO APPEAR ON THE OUTER PACKAGING AND THE IMMEDIATE 
PACKAGING 
 
CARTONS/ HDPE BOTTLE LABEL 
 
 
1. NAME OF THE MEDICINAL PRODUCT 
 
Raxone 150 mg film-coated tablets 
idebenone 
 
 
2. STATEMENT OF ACTIVE SUBSTANCE(S) 
 
Each film-coated tablet contains 150 mg of idebenone. 
 
 
3. LIST OF EXCIPIENTS 
 
Contains lactose and sunset yellow FCF (E110). See leaflet for further information. 
 
 
4. PHARMACEUTICAL FORM AND CONTENTS 
 
180 film-coated tablets  
 
 
5. METHOD AND ROUTE(S) OF ADMINISTRATION 
 
Read the package leaflet before use. 
 
For 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 
 
 



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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 
 
Santhera Pharmaceuticals (Deutschland) GmbH 
Marie-Curie Straße 8 
D-79539 Lörrach 
Germany 
 
 
12. MARKETING AUTHORISATION NUMBER(S)  
 
EU/1/15/1020/001 
 
 
13. BATCH NUMBER 
 
Batch  
 
 
14. GENERAL CLASSIFICATION FOR SUPPLY 
 
 
 
15. INSTRUCTIONS ON USE 
 
 
16. INFORMATION IN BRAILLE 
 
Raxone 150 mg 
 
17. UNIQUE IDENTIFIER – 2D BARCODE 
 
<2D barcode carrying the unique identifier included on the Outer Packaging.> 
 
18. UNIQUE IDENTIFIER – HUMAN READABLE DATA 
 
<PC {number} 
SN {number} 
NN {number} if applicable nationally > 
 
<Not applicable for the immediate packaging.>  



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B. PACKAGE LEAFLET 



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Package leaflet: Information for the user 
 

Raxone 150 mg film-coated tablets 
idebenone 

 
 

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 Raxone is and what it is used for  
2. What you need to know before you take Raxone  
3. How to take Raxone  
4. Possible side effects  
5. How to store Raxone  
6. Contents of the pack and other information 
 
 
1. What Raxone is and what it is used for 
 
Raxone contains a substance called idebenone.  
 
Idebenone is used to treat vision impairment in adults and adolescents with an eye disease called 
Leber’s Hereditary Optic Neuropathy (LHON).  
- This eye problem is inherited – this means it runs in families. 
- It is caused by a problem with your genes (called a “genetic mutation”) that affects the ability of 

cells in the eye to produce the energy they need to work normally, so they become inactive. 
- LHON can lead to loss of eyesight due to the inactivity of cells responsible for vision.  
 
Treatment with Raxone can restore the ability of cells to produce energy and so allow inactive eye 
cells to work again. This can lead to some improvement in lost eyesight.  
 
 
2. What you need to know before you take Raxone  
 
Do not take Raxone  
- if you are allergic to idebenone or any of the other ingredients of this medicine (listed in  
section 6).  
 
Warnings and precautions  
Talk to your doctor or pharmacist before taking Raxone if: 
- you have any blood, liver or kidney problems.  

 
Change in urine colour  
Raxone may make your urine become reddish brown. This change in colour is harmless – it does not 
mean your treatment needs to change. However, the change in colour could mean that you have 



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problems with your kidneys or bladder.  
- Tell your doctor if your urine changes colour. 
- He or she may do a urine check to make sure the change in colour is not hiding other problems. 
 
Tests 
Your doctor will check your eye-sight before you start taking this medicine and then at regular visits 
while you are taking it.  
 
Children and adolescents 
This medicine should not be used in children This is because it is not known if Raxone is safe or 
works in patients under 12 years of age. 
 
Other medicines and Raxone 
Some medicines may interact with Raxone. Tell your doctor if you are taking, have recently taken or 
might take any other medicines, especially any of the following: 
- antihistamines to treat allergies (astemizole, terfenadine) 
- to treat heartburn (cisapride) 
- to treat muscle and speech tics associated with Tourette syndrome (pimozide) 
- to treat hearth rhythm disorders (quinidine) 
- to treat migraine (dihydroergotamine, ergotamine) 
- to put you to sleep called “anaesthetics” (alfentanil) 
- to treat inflamation in rheumatoid arthritis and psoriasis (cyclosporine) 
- to prevent the rejection of an organ transplant (sirolimus, tacrolimus) 
- to treat strong pain called “opioids” (fentanyl) 

 
Pregnancy and breast-feeding  
If you are pregnant or breast-feeding, think you might be pregnant or are planning to have a baby, ask 
your doctor for advice before taking this medicine.  
- Your doctor will prescribe Raxone to you only if the benefits of the treatment are greater than 

the risks to the unborn child. 
- Raxone may pass into the mother’s milk. If you are breast-feeding your doctor will discuss with 

you whether to stop breast-feeding or to stop taking the medicine. This will take into account 
the benefit of breast-feeding to the child and the benefit of the medicine for you. 

 
Driving and using machines 
Raxone is not expected to affect your ability to drive or use machines.  
 
Raxone contains lactose and sunset yellow (E110) 
- Raxone contains lactose (a type of sugar). If you have been told by your doctor that you have an 

intolerance to some sugars, contact your doctor before taking this medicinal product.. 
- Raxone contains a colourant called “sunset yellow” (also called E110). This may cause allergic 

reactions. 
 
 
3. How to take Raxone 
 
Always take this medicine exactly as your doctor or pharmacist has told you. Check with your doctor 
or pharmacist if you are not sure.  
 
How much to take 
The recommended dose is 2 tablets three times a day - this is a total of 6 tablets per day.  

 
Taking this medicine 
- Take the tablets with food - this helps to get more of the medicine from your stomach into your 

blood. 
- Swallow the tablets whole with a glass of liquid. 
- Do not crush or chew the tablets. 



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- Take the tablets at the same time of day each day. For example in the morning at breakfast, with 
lunch at mid-day and with dinner in the evening. 

 
If you take more Raxone than you should 
If you take more Raxone than you should, talk to your doctor straight away. 
 
If you forget to take Raxone 
If you forget a dose, skip the missed dose. Take the next dose at the usual time. 
Do not take a double dose to make up for a forgotten dose.  
 
If you stop taking Raxone 
Talk to 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. The 
following side effect may happen with this medicine: 
 
Very common (may affect more than 1 in 10 people):  
- nasopharyngitis (cold) 
- cough 

 
Common (may affect up to 1 in 10 people):  
- diarrhoea (mild to moderate that usually does not require discontinuation of treatment) 
- back pain 

 
Unknown frequency (frequency cannot be estimated from the available data):  
- bronchitis 
- changes in blood test results: low level of white blood cells, or low level of red blood cells, or 

low level of platelets 
- increased cholesterol or fat in the blood –shown in tests 
- fits, feeling confused,  seeing or hearing things that are not real (hallucinations), feeling excited, 

movements that you cannot control, a tendency to wonder away, feeling dizzy, headache, 
feeling restless, dazed and unable to act or think normally 

- nausea, vomiting, loss of appetite, indigestion 
- high levels of some liver enzymes in the body which mean you have liver problems – shown in 

tests, high levels of “bilirubin” – this can make your skin and the whites of your eyes look 
yellow, hepatitis 

- rash, itching 
- pain in extremity 
- high levels of nitrogen in the blood - shown in tests change in urine colour 
- generally feeling unwell 
 
Reporting of side effects 
 
If you get any side effects, talk to your doctor. This includes any possible side effects not listed in this 
leaflet. You can also report side effects directly via the national reporting system in Appendix V. By 
reporting side effecs you can help provide more information on the safety of this medicine. 
 
 
5. How to store Raxone 
 
Keep this medicine out of the sight and reach of children. 
 



21 

Do not use this medicine after the expiry date which is stated on the carton and the bottle 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 Raxone contains 

- The active substance is idebenone. Each film-coated tablet contains 150 mg of idebenone.
- The other ingredients are:

Tablet core: lactose monohydrate, cellulose microcrystalline, croscarmellose sodium, povidone
K25, magnesium stearate and silica colloidal anhydrous.
Tablet film-coating: macrogol, poly(vinyl alcohol), talc, titanium dioxide, sunset yellow (E110).

What Raxone looks like and contents of the pack 

- Raxone film-coated tablets are orange, round tablets of 10 mm diameter, engraved with the
Santhera logo on one side and ‘150’ on the other side.

- Raxone is supplied in white plastic bottles. Each bottle contains 180 tablets.

Marketing Authorisation Holder and Manufacturer 

Santhera Pharmaceuticals (Deutschland) GmbH 
Marie-Curie Strasse 8 
79539 Lörrach 
Germany 
Tel: +49 (0) 7621 1690 200 
Fax: +49 (0) 7621 1690 201 
Email: office@santhera.com 

This leaflet was last revised in 

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 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. There are also links to other websites about rare diseases and treatments. 



Annex IV 

Grounds for one additional renewal 

22



Grounds for one additional renewal 

Based upon the data that have become available since the granting of the initial Marketing authorisation, 
the CHMP considers that the benefit-risk balance of Raxone remains positive, but recommended that one 
additional five-year renewal be required because two key SOBs need to be fulfilled with this MA under 
exceptional circumstances: 

Two SOBs have to be fulfilled: 

Number Description Due date 

SOB 015 
(former SOB-
1)  

Open-label study to assess the efficacy and safety of Raxone in LHON 
patients (LEROS) (SNT-IV-005) 

Primary: 
- To assess the effectiveness of Raxone, compared with no treatment,
in improving vision or in preventing its worsening, in patients treated
with Raxone less than 1 year after the symptoms appear.

Secondary: 
- To assess the effectiveness of Raxone, compared with no treatment,
in improving vision or in preventing its worsening in patients treated
with Raxone more than 1 year after the symptoms appear.

- To compare the effectiveness of Raxone in improving vision or in
preventing its worsening in patients treated with Raxone less than 1
year and more than 1 year after the symptoms appear.
- To assess the influence of mutation on improving vision or preventing
its worsening in LHON patients treated with Raxone.
- To assess if the effectiveness of Raxone changes depending on the
duration between appearance of symptoms and start of Raxone
treatment.
- To assess the influence of duration of treatment with Raxone on
changes in visual acuity in LHON patients.
- To assess safety of long-term treatment of LHON patients with
Raxone

Interim reports 
to be provided 
in annual re-
assessments 
Final study 
report: 
31 August 
2021 
(to be 
submitted with 
the annual re-
assessment) 

SOB 003 A non-interventional study of clinical experience in patients prescribed 
Raxone for the treatment of LHON 

Primary: 
- To further evaluate the long-term safety profile of Raxone in the
treatment of LHON when used in routine clinical practice.

Secondary: 
- To further evaluate the long-term effectiveness of Raxone in the
treatment of LHON when used in routine clinical practice.
- To quantify discontinuation of treatment due to adverse events or
due to lack of therapeutic response or loss of response.
- To further understand the risk of abnormal liver function tests and
hepatitis (PASS registry SNT-IV-003) (PAROS)

Interim reports 
and final study 
report to be 
submitted with 
annual re-
assessments 

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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
	Grounds for one additional renewal