PTC Therapeutics (PTCT) Receives EMA CHMP Approval for Translarna Marketing Authorization in mmDMD

November 11, 2016 7:17 AM EST

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PTC Therapeutics, Inc. (Nasdaq: PTCT) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (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. In connection with the renewal, the marketing authorization will include a specific obligation to conduct an additional long-term post-authorization trial.

"We are pleased with this outcome which took into account all available data for Translarna," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "This decision reflects the benefit that Translarna is having for patients suffering from nonsense mutation Duchenne muscular dystrophy."

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.

"Translarna has shown clinically meaningful benefits for patients," said Eugenio Mercuri, M.D., Professor of Pediatric Neurology at the Catholic University, Rome, Italy. "Duchenne is a devastating disease with a progressive loss of function. Maintaining function is of the utmost importance to patients."

"The consistency of Translarna's benefit shown across key endpoints is impressive for a dystrophin replacement therapy," said Craig McDonald, M.D., Professor of Pediatrics and Chair of the Department of Physical Medicine & Rehabilitation at University of California. "I am encouraged for the DMD community by the CHMP's recommendation."

The CHMP has requested that PTC conduct a new 18-month randomized, placebo-controlled study in patients with nonsense mutation Duchenne muscular dystrophy, 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.

"For boys with Duchenne, every day matters and functional loss cannot be regained. Patients need access to innovative new therapies like Translarna," stated Filippo Buccella, founder of the Italian Parent Project, a patient advocacy group for Duchenne Muscular Dystrophy.

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