This medicine is authorised for use in the European Union.


Translarna is a medicine that is used to treat patients aged 2 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 patients whose disease is caused by a specific genetic defect (called a ‘nonsense mutation’) in the dystrophin gene.

Duchenne muscular dystrophy is rare, and Translarna was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 27 May 2005.

This EPAR was last updated on 25/04/2023

Authorisation details

Product details
Agency product number
Active substance
International non-proprietary name (INN) or common name
Therapeutic area (MeSH)
Muscular Dystrophy, Duchenne
Anatomical therapeutic chemical (ATC) code
Additional monitoringAdditional monitoring

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

Conditional approvalConditional approval

This medicine received a conditional marketing authorisation. This was granted in the interest of public health because the medicine addresses an unmet medical need and the benefit of immediate availability outweighs the risk from less comprehensive data than normally required. For more information, see Conditional marketing authorisation.


This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

Publication details
Marketing-authorisation holder
PTC Therapeutics International Limited
Date of issue of marketing authorisation valid throughout the European Union
Contact address
5th Floor, 3 Grand Canal Plaza
Grand Canal Street Upper
Dublin 4
D04 EE70

Product information

16/03/2023 Translarna - EMEA/H/C/002720 - II/0068

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Product information documents contain:

You can find product information documents for centrally authorised human medicines on this website. For centrally authorised veterinary medicines authorised or updated from February 2022, see the Veterinary Medicines Information website.

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 2 years and older. Efficacy has not been demonstrated in non-ambulatory patients.

The presence of a nonsense mutation in the dystrophin gene should be determined by genetic testing.

Assessment history

Changes since initial authorisation of medicine


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