US-based pharmaceutical company PTC Therapeutics, Inc. (NASDAQ: PTCT) announced on Friday that the European Commission (EC) has adopted the opinion of the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) to not renew the authorisation of Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy.

While the opinion effectively removes the drug's conditional marketing authorisation in the European Economic Area, the EC indicated that individual countries within the European Union can leverage Articles 117(3) and 5(1) of the EU Directive 2001/83 to allow continued use of Translarna.

The product was discovered and developed by PTC Therapeutics and is a protein restoration therapy aimed at allowing the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation.

Matthew C Klein, MD, PTC Therapeutics chief executive officer, said, 'We are of course disappointed that after this prolonged period of review the European Commission has decided to adopt the CHMP negative opinion on Translarna. The EC's indication that European Union member states have a mechanism to maintain treatment speaks to the safety, benefit and lack of alternative therapies for boys and young men with nonsense mutation Duchenne muscular dystrophy. We look forward to working on a country-by-country basis to provide commercial drug where possible.'