CHMP changes mind on PTC Therapeutics' Duchenne drug Translarna, giving a positive opinion

25 May 2014

In something of a surprise for PTC Therapeutics (Nasdaq: PTCT) and observers, the European Medicines Agency’s advisory panel has reversed an earlier negative opinion of the company’s drug for the treatment of Duchenne muscular dystrophy (The Pharma Letter January 24), causing the firm’s stock to rocket more than 30% to $20.03 on Friday, having rise by as much as 52.7%.

Following PTC’s request for re-examination, the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion regarding the company's application for a conditional marketing authorization of Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged five years and older.

If Translarna gets conditional approval from the European Commission, PTC Therapeutics will still have to complete clinical studies of the drug. The conditional approval must be renewed annually until full approval is granted.

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