Translarna
ataluren
Table of contents
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.
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Translarna : EPAR - Medicine overview (PDF/110.07 KB)
First published: 04/09/2014
Last updated: 31/08/2018 -
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Translarna : EPAR - Risk-management-plan summary (PDF/250.3 KB)
First published: 30/07/2019
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 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 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. |
Orphan |
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 | |
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Marketing-authorisation holder |
PTC Therapeutics International Limited
|
Revision |
22
|
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
16/03/2023 Translarna - EMEA/H/C/002720 - II/0068
This medicine’s product information is available in all official EU languages.
Select ‘available languages’ to access the language you need.
Product information documents contain:
- summary of product characteristics (annex I);
- manufacturing authorisation holder responsible for batch release (annex IIA);
- conditions of the marketing authorisation (annex IIB);
- labelling (annex IIIA);
- package leaflet (annex IIIB).
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
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
News
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13/10/2023
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15/09/2023
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15/09/2023
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Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 14-17 October 201918/10/2019
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26/07/2019
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28/06/2019
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01/06/2018
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24/03/2017
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14/03/2017
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29/09/2014
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10/07/2014
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23/05/2014
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23/05/2014
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21/02/2014
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Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 20-23 January 201424/01/2014