Translarna™ (ataluren) Market Authorisation Not Renewed by European Commission.

The European Commission (EC) has announced that it will not renew the market authorisation of Translarna™ (ataluren) for treating nonsense mutation Duchenne muscular dystrophy. After re-examining the data, the EMA’s human medicines committee (CHMP) concluded that the effectiveness of Translarna could not be confirmed.  

While this action effectively removes the drug's conditional marketing authorisation in the Republic of Ireland and other European countries, the European Commission has indicated that individual countries within the European Union can leverage Articles 117(3) and 5(1) of the EU Directive 2001/83 to allow the continued use of Translarna.

PTC Therapeutics plans to work on a country-by-country basis to provide the drug commercially where possible. We encourage those impacted by this decision to speak with their clinician about the option to apply for continued use through the EU Directive 2001/83.

MDI recognises that this decision is likely to cause feelings of disappointment, worry, and frustration, for those affected. Families in Ireland campaigned for years to get access to this treatment and have lived with uncertainty and concern over the past 18 months while waiting for the European Commission’s decision.

If you would like to speak to a member of staff at MDI, you can reach us by calling the Support and Advocacy phoneline at 01 6236414, connecting via our website, or emailing MDI’s research officer at mdiresearch@mdi.ie 

To read PTCs full statement click here.