Translarna

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ataluren

Authorised
This medicine is authorised for use in the European Union.

Overview

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 13/06/2019

Authorisation details

Product details
Name
Translarna
Agency product number
EMEA/H/C/002720
Active substance
Ataluren
International non-proprietary name (INN) or common name
ataluren
Therapeutic area (MeSH)
Muscular Dystrophy, Duchenne
Anatomical therapeutic chemical (ATC) code
M09AX03
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.

OrphanOrphan

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
Revision
11
Date of issue of marketing authorisation valid throughout the European Union
31/07/2014
Contact address
5th Floor, 3 Grand Canal Plaza
Grand Canal Street Upper
Dublin 4
D04 EE70
Ireland

Product information

09/04/2019 Translarna - EMEA/H/C/002720 - IAIN/0052/G

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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

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