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.
Translarna : EPAR - Medicine overview (PDF/110.07 KB)
First published: 04/09/2014
Last updated: 31/08/2018
Translarna : EPAR - Risk-management-plan summary (PDF/250.3 KB)
First published: 30/07/2019
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|International non-proprietary name (INN) or common name||
|Therapeutic area (MeSH)||
Muscular Dystrophy, Duchenne
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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.
PTC Therapeutics International Limited
|Date of issue of marketing authorisation valid throughout the European Union||
25/07/2019 Translarna - EMEA/H/C/002720 - R/0051
- 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
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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.
European Medicines Agency recommends 39 medicines for human use for marketing authorisation in first half of 201410/07/2014
European Medicines Agency recommends first-in-class medicine for treatment of Duchenne muscular dystrophy23/05/2014
Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 17-20 February 201421/02/2014
Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 20-23 January 201424/01/2014