Vertex Pharmaceuticals Inc.‘s (VRTX) Kalydeco (ivacaftor) recently scored a win when the US Food and Drug Administration (FDA) granted the company’s new drug application (NDA) a priority review status. The regulatory body has set a target date of April 18, 2012, for the drug’s NDA, submitted in October 2011.
Vertex Pharma is seeking approval of Kalydeco for treating patients (aged 6 years and above) suffering from cystic fibrosis (CF).The patients should have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
We note that the US regulatory authority generally reviews those drugs on a priority basis which offer advances in treating diseases that do not have adequate therapy. Applications for priority review designated drugs are reviewed by the FDA within six months of submission as against the usual 10 months.
Moreover, Vertex Pharma’s endeavor to get Kalydeco approved in the European Union is on track. The European Medicines Agency (EMA) has granted accelerated assessment status to the marketing authorization application (MAA) for Kalydeco, which has brought down the review time of the application to 150 days from 210 days.
The regulatory submissions made by Vertex Pharma were based on data from two late-stage studies, STRIVE and ENVISION. The trial results demonstrated that patients (with CF who had at least one copy of the G551D mutation) treated with Kalydeco, experienced rapid, significant and sustained improvements across multiple disease measures, including lung function, compared to those in the placebo arm.
We believe that the approval of Kalydeco as a treatment for CF would be a major boost for the company, as currently there are hardly any treatments in the market for the indication.
We note that it has been a good month so far for Vertex Pharma, with two of its pipeline candidates making progress. Last week, Vertex Pharma along with partner Alios BioPharma, Inc. announced the initiation of two early-stage trials with candidates ALS-2200 and ALS-2158. The companies are studying the two candidates as a treatment for chronic genotype-1 hepatitis C virus (HCV). Data from the trials are expected in the second quarter of 2012.
We currently have a Neutral recommendation on Vertex Pharma. The stock carries a Zacks #2 Rank (Buy rating) in the short-run.
