Translarna

ataluren

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This is a summary of the European public assessment report (EPAR) for Translarna. It explains how the Agency assessed the medicine to recommend its authorisation in the EU and its conditions of use. It is not intended to provide practical advice on how to use Translarna.

For practical information about using Translarna, patients should read the package leaflet or contact their doctor or pharmacist.

What is Translarna and what is it used for?

Translarna is a medicine that contains the active substance ataluren. It is used to treat patients aged 5 years and older with Duchenne muscular dystrophy who are able to walk. Duchenne muscular dystrophy is a genetic disease that gradually causes weakness and loss of muscle function. Translarna is used in the small group of Duchenne patients whose disease is caused by a specific genetic defect (called a ‘nonsense mutation’) in the dystrophin gene.

Because the number of patients with Duchenne muscular dystrophy is low, the disease is considered ‘rare’, and Translarna was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 27 May 2005.

How is Translarna used?

Translarna can only be obtained with a prescription and treatment should be started by a specialist doctor experienced in the management of Duchenne/Becker muscular dystrophy.

Before starting treatment with Translarna, patients will have a blood test done to confirm that their disease is due to a nonsense mutation and that they are therefore suitable for treatment with Translarna.

Translarna is available as granules (100, 250 and 1,000 mg) to be taken by mouth after mixing them with liquid or semi-solid food (such as yogurt). Translarna is taken three times a day, and the recommended dose is 10 mg/kg (10 mg per kilogram body weight) in the morning, 10 mg/kg at midday and 20 mg/kg in the evening (making a total daily dose of 40 mg/kg). For further information, see the package leaflet.

How does Translarna work?

Patients with Duchenne muscular dystrophy lack normal dystrophin, a protein found in muscles. Because this protein helps to protect muscles from injury as muscles contract and relax, in patients with Duchenne muscular dystrophy the muscles become damaged and eventually stop working.

Duchenne muscular dystrophy can be caused by a number of genetic abnormalities. Translarna is for use in patients whose disease is due to the presence of certain defects (called nonsense mutations) in the dystrophin gene which prematurely stop the production of a normal dystrophin protein, leading to a shortened dystrophin protein that does not function properly. Translarna works in these patients by enabling the protein-making apparatus in cells to move past the defect, allowing the cells to produce a functional dystrophin protein.

What benefits of Translarna have been shown in studies?

Translarna has been studied in one main study involving 174 patients with Duchenne muscular dystrophy who were able to walk, where two doses of Translarna (40 mg/kg daily and 80 mg/kg daily) were compared with placebo (a dummy treatment). The main measure of effectiveness was the change in the distance the patient could walk in six minutes after 48 weeks of treatment.

Although an initial analysis of the results of all the data from the study did not show a significant difference in the distances patients in the Translarna and placebo groups could walk, further analyses indicated that walking ability reduced to a lesser extent with 40 mg/kg daily Translarna than with placebo: after 48 weeks of treatment patients receiving 40 mg/kg daily Translarna could walk on average 31.3 metres more than those given placebo. This beneficial effect of the lower dose was also supported by improvements in other measures of effectiveness, including those directly linked to patients’ daily activities. No improvement was seen with the higher dose (80 mg/kg/day).

What are the risks associated with Translarna?

The most common side effects with Translarna (which may affect more than 1 in 10 people) are nausea (feeling sick), vomiting and headache.

Translarna must not be used at the same time as certain antibiotics known as aminoglycosides when these are given by injection into a vein.

For the full list of all side effects and restrictions with Translarna, see the package leaflet.

Why is Translarna approved?

The Agency’s Committee for Medicinal Products for Human Use (CHMP) decided that Translarna’s benefits are greater than its risks and recommended that it be approved for use in the EU.

Despite the limited data available, and the fact that no benefit was demonstrated at a higher dose of 80 mg/kg daily, the CHMP considered that there is some evidence that Translarna 40 mg/kg daily slows the progression of the disease and that its safety profile is not of major concern. The Committee also acknowledged the seriousness of Duchenne muscular dystrophy and the unmet medical need of patients with this condition.

Translarna has been given ‘conditional approval’. This means that there is more evidence to come about the medicine, which the company is required to provide. Every year, the European Medicines Agency will review any new information that becomes available and this summary will be updated as necessary.

What information is still awaited for Translarna?

Since Translarna has been granted a conditional approval, the company that markets Translarna shall provide further data on the effectiveness and safety of the medicine from an ongoing confirmatory study in DMD patients with the nonsense mutation.

What measures are being taken to ensure the safe and effective use of Translarna?

A risk management plan has been developed to ensure that Translarna is used as safely as possible. Based on this plan, safety information has been included in the summary of product characteristics and the package leaflet for Translarna, including the appropriate precautions to be followed by healthcare professionals and patients.

Further information can be found in the summary of the risk management plan.

Other information about Translarna

The European Commission granted a marketing authorisation valid throughout the European Union for Translarna on 31 July 2014.

For more information about treatment with Translarna, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.

Name Language First published Last updated
Translarna : EPAR - Summary for the public BG = bălgarski 2014-09-04  
Translarna : EPAR - Summary for the public ES = español 2014-09-04  
Translarna : EPAR - Summary for the public CS = čeština 2014-09-04  
Translarna : EPAR - Summary for the public DA = dansk 2014-09-04  
Translarna : EPAR - Summary for the public DE = Deutsch 2014-09-04  
Translarna : EPAR - Summary for the public ET = eesti keel 2014-09-04  
Translarna : EPAR - Summary for the public EL = elliniká 2014-09-04  
Translarna : EPAR - Summary for the public EN = English 2014-09-04  
Translarna : EPAR - Summary for the public FR = français 2014-09-04  
Translarna : EPAR - Summary for the public IT = italiano 2014-09-04  
Translarna : EPAR - Summary for the public LV = latviešu valoda 2014-09-04  
Translarna : EPAR - Summary for the public LT = lietuvių kalba 2014-09-04  
Translarna : EPAR - Summary for the public HU = magyar 2014-09-04  
Translarna : EPAR - Summary for the public MT = Malti 2014-09-04  
Translarna : EPAR - Summary for the public NL = Nederlands 2014-09-04  
Translarna : EPAR - Summary for the public PL = polski 2014-09-04  
Translarna : EPAR - Summary for the public PT = português 2014-09-04  
Translarna : EPAR - Summary for the public RO = română 2014-09-04  
Translarna : EPAR - Summary for the public SK = slovenčina 2014-09-04  
Translarna : EPAR - Summary for the public SL = slovenščina 2014-09-04  
Translarna : EPAR - Summary for the public FI = suomi 2014-09-04  
Translarna : EPAR - Summary for the public SV = svenska 2014-09-04  
Translarna : EPAR - Summary for the public HR = Hrvatski 2014-09-04  
Name Language First published Last updated
Translarna : EPAR - Risk-management-plan summary (English only) 2014-09-04  

This EPAR was last updated on 14/01/2016 .

Authorisation details

Product details

Product details for Translarna
NameTranslarna
Agency product numberEMEA/H/C/002720
Active substance

ataluren

International non-proprietary name (INN) or common name

ataluren

Therapeutic area Muscular Dystrophy, Duchenne
Anatomical therapeutic chemical (ATC) code
Additional monitoring

This medicine is under additional monitoring. This means that it is being monitored even more intensively than other medicines. For more information, see medicines under additional monitoring.

Treatment of rare diseases

This medicine has an "orphan designation" which means that it is used to treat life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in the European Union, or are medicines which, for economic reasons, would be unlikely to be developed without incentives.

Conditional Approval

Sometimes, the CHMP recommends that a medicine be given ‘conditional approval’. This happens when the Committee has based its positive opinion on data which, while not yet comprehensive, indicate that the medicine’s benefits outweigh its risks.

The company is given obligations to fulfil, such as the performance of further studies. The approval is renewed on a yearly basis until all obligations have been fulfilled, and is then converted from a conditional approval into a normal approval. Conditional approvals can only be granted for medicines that satisfy an ‘unmet medical need’, meaning the medicine is intended to be used for a disease or condition for which no treatment is readily available, and it is therefore important that patients have early access to the medicine concerned.

Publication details

Publication details for Translarna
Marketing-authorisation holder

PTC Therapeutics International Limited

Revision3
Date of issue of marketing authorisation valid throughout the European Union31/07/2014

Contact address:

PTC Therapeutics International Limited
77 Sir John Rogerson's Quay
Dublin 2
Ireland

Product information

Product information

17/12/2015  Translarna -EMEA/H/C/002720 -IB/0015

Name Language First published Last updated
Translarna : EPAR - Product Information EN = English 2014-09-04 2016-01-14
Translarna : EPAR - Product Information EN = English 2014-09-04 2016-01-14
Translarna : EPAR - Product Information EN = English 2014-09-04 2016-01-14
Translarna : EPAR - Product Information EN = English 2014-09-04 2016-01-14
Translarna : EPAR - Product Information EN = English 2014-09-04 2016-01-14
Translarna : EPAR - Product Information EN = English 2014-09-04 2016-01-14
Translarna : EPAR - Product Information EN = English 2014-09-04 2016-01-14
Translarna : EPAR - Product Information EN = English 2014-09-04 2016-01-14
Translarna : EPAR - Product Information EN = English 2014-09-04 2016-01-14
Translarna : EPAR - Product Information EN = English 2014-09-04 2016-01-14
Translarna : EPAR - Product Information EN = English 2014-09-04 2016-01-14
Translarna : EPAR - Product Information EN = English 2014-09-04 2016-01-14
Translarna : EPAR - Product Information EN = English 2014-09-04 2016-01-14
Translarna : EPAR - Product Information EN = English 2014-09-04 2016-01-14
Translarna : EPAR - Product Information EN = English 2014-09-04 2016-01-14
Translarna : EPAR - Product Information EN = English 2014-09-04 2016-01-14
Translarna : EPAR - Product Information EN = English 2014-09-04 2016-01-14
Translarna : EPAR - Product Information EN = English 2014-09-04 2016-01-14
Translarna : EPAR - Product Information EN = English 2014-09-04 2016-01-14
Translarna : EPAR - Product Information EN = English 2014-09-04 2016-01-14
Translarna : EPAR - Product Information EN = English 2014-09-04 2016-01-14
Translarna : EPAR - Product Information EN = English 2014-09-04 2016-01-14
Translarna : EPAR - Product Information EN = English 2014-09-04 2016-01-14
Translarna : EPAR - Product Information EN = English 2014-09-04 2016-01-14
Translarna : EPAR - Product Information EN = English 2014-09-04 2016-01-14

Contents

  • Annex I - Summary of product characteristics
  • Annex IIA - Manufacturing-authorisation holder responsible for batch release
  • Annex IIB - Conditions of the marketing authorisation
  • Annex IIIA - Labelling
  • Annex IIIB - Package leaflet

Please note that the size of the above document can exceed 50 pages.

You are therefore advised to be selective about which sections or pages you wish to print.


Name Language First published Last updated
Translarna : EPAR - All Authorised presentations EN = English 2014-09-04  
Translarna : EPAR - All Authorised presentations EN = English 2014-09-04  
Translarna : EPAR - All Authorised presentations EN = English 2014-09-04  
Translarna : EPAR - All Authorised presentations EN = English 2014-09-04  
Translarna : EPAR - All Authorised presentations EN = English 2014-09-04  
Translarna : EPAR - All Authorised presentations EN = English 2014-09-04  
Translarna : EPAR - All Authorised presentations EN = English 2014-09-04  
Translarna : EPAR - All Authorised presentations EN = English 2014-09-04  
Translarna : EPAR - All Authorised presentations EN = English 2014-09-04  
Translarna : EPAR - All Authorised presentations EN = English 2014-09-04  
Translarna : EPAR - All Authorised presentations EN = English 2014-09-04  
Translarna : EPAR - All Authorised presentations EN = English 2014-09-04  
Translarna : EPAR - All Authorised presentations EN = English 2014-09-04  
Translarna : EPAR - All Authorised presentations EN = English 2014-09-04  
Translarna : EPAR - All Authorised presentations EN = English 2014-09-04  
Translarna : EPAR - All Authorised presentations EN = English 2014-09-04  
Translarna : EPAR - All Authorised presentations EN = English 2014-09-04  
Translarna : EPAR - All Authorised presentations EN = English 2014-09-04  
Translarna : EPAR - All Authorised presentations EN = English 2014-09-04  
Translarna : EPAR - All Authorised presentations EN = English 2014-09-04  
Translarna : EPAR - All Authorised presentations EN = English 2014-09-04  
Translarna : EPAR - All Authorised presentations EN = English 2014-09-04  
Translarna : EPAR - All Authorised presentations EN = English 2014-09-04  
Translarna : EPAR - All Authorised presentations EN = English 2014-09-04  
Translarna : EPAR - All Authorised presentations EN = English 2014-09-04  

Pharmacotherapeutic group

Other drugs for disorders of the musculo-skeletal system

Therapeutic indication

Translarna is indicated for the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 5 years and older.

Assessment History

Changes since initial authorisation of medicine

Name Language First published Last updated
Translarna : EPAR - Procedural steps taken and scientific information after authorisation EN = English 2014-11-28 2016-01-14

Initial marketing-authorisation documents

Name Language First published Last updated
Translarna : EPAR - Public assessment report EN = English 2014-09-04  
CHMP summary of positive opinion for Translarna EN = English 2014-05-23