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Vertex Pharmaceuticals' Kalydeco Wins Positive EU Opinion

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Vertex Pharmaceuticals’ (VRTX - Free Report) cystic fibrosis (CF) drug, Kalydeco (ivacaftor) won a positive opinion from the European Committee for Medicinal Products for Human Use (CHMP) for use in CF patients (6 years of age and above) with one of eight non-G551D gating mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Kalydeco is already approved in the EU for CF patients (6 years of age and above) who have at least one copy of the G551D mutation – this is the most common gating mutation. Approval for the additional eight gating mutations will provide access to about 250 eligible patients. Approval in the EU for the additional gating mutations could come in the September-October timeframe. Vertex Pharma had gained FDA approval for the eight additional mutations earlier this year in Feb.

Meanwhile, Vertex Pharma also received a positive opinion from the CHMP regarding the inclusion of long-term data from the phase III PERSIST study. PERSIST, an open-label, 96-week, rollover extension study, showed that Kalydeco’s safety and efficacy seen in the phase III STRIVE and ENVISION studies was maintained through nearly three years in G551D patients.

We are encouraged by Vertex Pharma’s continued progress with its CF franchise. With the company deciding to exit its hepatitis C franchise, Vertex Pharma is banking on the CF franchise for growth. The company was in the news last week when it presented highly-awaited phase III data from studies evaluating Kalydeco in combination with lumacaftor. Vertex Pharma intends to file for approval of this combination in the U.S. and EU in the fourth quarter for people with CF (12 years and older) who have two copies of the F508del mutation.

Vertex Pharma is a Zacks Rank #3 (Hold) stock. Some better-ranked stocks in the health care sector include Biogen (BIIB - Free Report) , Actelion and Regeneron Pharmaceuticals (REGN - Free Report) – all three are Zacks Rank #1 (Strong Buy) stocks.

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