Translarna

ataluren

Opinion

Update of 25 July 2019:
The applicant for Translarna has requested a re-examination of EMA’s June 2019 opinion. Upon receipt of the grounds of the request, the Agency will re-examine its opinion and issue a final recommendation.

The European Medicines Agency has recommended the refusal of a change to the marketing authorisation for Translarna (ataluren) to add the treatment of patients with Duchenne muscular dystrophy who are no longer able to walk.

The Agency issued this opinion on 27 June 2019.

The company that applied for the change to the authorisation, PTC Therapeutics International Limited, may ask for re-examination of the opinion within 15 days of receiving the opinion.
 

Key facts

Name
Translarna
Agency product number
EMEA/H/C/002720
International non-proprietary name (INN) or common name
  • ataluren
Active substance
  • Ataluren
Therapeutic area
Muscular Dystrophy, Duchenne
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.

Date opinion adopted
27/06/2019
Company name
PTC Therapeutics International Limited
Status
Negative
Application type
Post-authorisation

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