Market Overview

PTC Therapeutics Spikes 40% After Translarna Update In England


Shares of PTC Therapeutics, Inc. (NASDAQ: PTCT), a biopharmaceutical company that focuses on therapeutics that target post-transcriptional control processes, surged higher by more than 40 percent Friday afternoon.

Investors and traders were reacting to the company's announcement that the England-based National Institute for Health and Care Excellence (NICE) has recommended the company's Translarna (ataluren) for ambulatory patients that suffer from nonsense mutation Duchenne muscular dystrophy (nmDMD).

Translarna was approved by the European Commission in August 2014 to treat nmDMD and is already available in 23 countries.

PTC Therapeutics is currently working with England's medical authorities (NHS England) to finalize a Managed Access Agreement (MAA) outlining financial and clinical details surrounding the use of Translarna. The MAA will allow the company to collect further data on the efficacy of Translarn for the treatment of nmDMD over a five-year period.

"We are extremely pleased by the NICE recommendation, which recognizes Translarna as an innovative medicine with the potential to change the course of this devastating disease," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "This is a crucial step for the boys and young men in England with nonsense mutation DMD. We are grateful to the patients, families, advocacy groups and physicians who have supported PTC Therapeutics through this important access process and look forward to working with NHS England to conclude the managed access agreement."

"This is an important day for the Duchenne community, which has been working hard to make this ground-breaking drug available to boys with nonsense mutation Duchenne muscular dystrophy," said Paul Lenihan, Chief Executive of Action Duchenne. "We are delighted by this positive recommendation and NICE's recognition that Translarna is an important new medicine for patients. This decision is a hugely encouraging sign that both NICE and NHS England have listened to the patient community, bringing hope to each and every parent and patient fighting DMD."

Posted-In: Biopharmaceutical Companies Biopharmaceutical Stocks Duchenne Musclur Dystrophy nmDMD PTCT TherapeuticsBiotech News General


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