Early Approval for Vertex CF Drug


Vertex Pharmaceuticals Inc.’s (NasdaqGS:VRTX - News) shares jumped over 6% buoyed by news of the US Food and Drug Administration approving its lead pipeline candidate, Kalydeco (ivacaftor), for treating patients (aged 6 years and above) suffering from cystic fibrosis (NYSE:CF - News).

The approval is for the treatment of patients who have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator (:CFTR) gene.

We note that in December last year, the regulatory body had granted priority review status to the company’s new drug application (:NDA) for Kalydeco, and had set a target date of April 18, 2012. However, the FDA approved the drug within three months of the NDA filing (October 2011), as there are not adequate therapies in the market for treating CF.

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 (NYSE:MAA - News) for Kalydeco, which has brought down the review time for 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.

Our View

We currently have a Neutral recommendation on Vertex Pharma. The stock carries a Zacks #3 Rank (Hold rating) in the short-run. We believe that the approval of Kalydeco was highly awaited at Vertex Pharma, as the company currently markets only one drug, Incivek (telaprevir), approved in the US in May 2011 for the treatment of hepatitis C virus (:HCV), in both treatment-naïve and treatment-failed patients.

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