PTC Therapeutics Up on Positive CHMP Opinion - Analyst Blog

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) provided a positive opinion on the marketing authorization of PTC Therapeutics, Inc.'s PTCT Translarna. Shares of PTC Therapeutics shot up around 30.7% following the news.

PTC Therapeutics is looking for a conditional approval for Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients of five years and above. We note that the conditional marketing authorization will allow the company to market the product with unified labelling in the 28 countries that are Member States of the EU, as well as European Economic Area members Iceland, Liechtenstein and Norway.

A final decision from the European Commission on the approval of Translarna is expected by Aug 2014. The positive opinion from the CHMP is a huge relief for the company. In Jan 2014, the EMA advisory body had provided a negative opinion on the approval of Translarna. The company had then requested the CHMP for a re-examination of the opinion.

We note that the favorable opinion from the CHMP was based on positive data from a phase IIb, double-blind, placebo controlled study on Translarna. Results from the study revealed that patients with nmDMD under Translarna treatment walked an average of 31.3 meters more than patients under placebo at week 48. Translarna treated patients demonstrated a slower rate of decline in ambulation as well. Translarna was also well tolerated during the study.

PTC Therapeutics is currently running a confirmatory, phase III, ACT DMD study on Translarna. The company intends to complete enrollment for the study by mid 2014. PTC Therapeutics believes that results from the study will help them to gain full EU approval of Translarna. The company also intends to file for marketing approval application in the U.S. on the basis of the results from the ACT DMD study.

We are encouraged by the positive opinion from the CHMP on the approval of Translarna. We expect investors to stay focused on the company's progress with Translarna going forward. The successful development of Translarna is crucial for the company.

PTC Therapeutics, a biopharma company, presently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the same sector include Gilead Sciences Inc. GILD, Alexion Pharmaceuticals, Inc. ALXN and AVEO Pharmaceuticals, Inc. AVEO. While Gilead carries a Zacks Rank #1 (Strong Buy), Alexion and AVEO hold a Zacks Rank #2 (Buy).

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