EMA — authorised 31 July 2014
- Marketing authorisation holder: PTC Therapeutics International Limited
- Status: approved
🇪🇺 Translarna in European Union
EMA authorised Translarna on 31 July 2014
Marketing authorisations
EMA — authorised 31 July 2014
- Application: EMEA/H/C/002720
- Marketing authorisation holder: PTC Therapeutics International Limited
- Local brand name: Translarna
- 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.
- Pathway: conditional
- Status: withdrawn
Translarna in other countries
Same drug, different regulators. Each page below lists the local marketing authorisation, HTA position, pricing, and adverse-event data.
Other Rare Disease approved in European Union
Frequently asked questions
Is Translarna approved in European Union?
Yes. EMA authorised it on 31 July 2014; EMA authorised it on 31 July 2014.
Who is the marketing authorisation holder for Translarna in European Union?
PTC Therapeutics International Limited holds the EU marketing authorisation.