PTC Therapeutics Inc. (NASDAQ: PTCT) shares skyrocketed early on Friday after the company received positive news from the European Medicines Agency (EMA). The company announced that the Committee for Medicinal Products for Human Use (CHMP) of the EMA has recommended the renewal of the conditional marketing authorization of Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients five years and older.

Also in connection with the renewal, the marketing authorization will include a specific obligation to conduct an additional long-term post-authorization trial.

Generally speaking, the CHMP opinion forms the basis for a European Commission decision on the renewal of the marketing authorization. The European Commission generally delivers its decision within three months.

The CHMP has requested that PTC conduct a new 18-month randomized, placebo-controlled study in patients with nmDMD, as a specific post-authorization obligation, with results expected to be available in the first quarter of 2021. This study will be followed by an 18-month open-label extension period where all patients will be switched to Translarna. PTC has proposed a trial similar in size to ACT DMD and details of the protocol are expected to be finalized in future interactions with the EMA. Conditional marketing authorizations are subject to annual reassessment and renewal.

Craig McDonald, M.D., Professor of Pediatrics and Chair of the Department of Physical Medicine & Rehabilitation at University of California, commented:

The consistency of Translarna’s benefit shown across key endpoints is impressive for a dystrophin replacement therapy. I am encouraged for the DMD community by the CHMP’s recommendation.

Eugenio Mercuri, M.D., Professor of Pediatric Neurology at the Catholic University, Rome, Italy, added:

Translarna has shown clinically meaningful benefits for patients. Duchenne is a devastating disease with a progressive loss of function. Maintaining function is of the utmost importance to patients.

Shares of PTC Therapeutics closed Thursday up 7% at $6.00, with a consensus analyst price target of $11.13 and a 52-week trading range of $4.03 to $35.40. Following the update, the stock was up about 80% at $10.83 in early trading indications Friday.

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