Zacks_Analysts' Blog : Vertex Seeks CF Drug Approval - Analyst Blog

Vertex Pharmaceuticals Inc. (VRTX) recently announced the submission of a new drug application (NDA) to the US Food and Drug Administration (FDA), seeking approval to market Kalydeco (VX-770, ivacaftor). Upon approval, the drug will be used to treat 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.

The company has also requested the regulatory body to review the application on a priority basis. We note that if priority review status is granted by the FDA, the NDA will be reviewed within six months, instead of the normal 10 months.

Additionally, Vertex Pharma intends to submit a marketing authorization application (MAA) for Kalydeco with the European Medicines Agency (EMA) by the end of October 2011. The EMA has accepted the company’s request for accelerated assessment, which will bring down the review time from 210 days to 150 days following the commencement of the review.

We note that the company is currently studying Kalydeco in combination with VX-809, as a treatment for CF. In June 2011, Vertex Pharma reported positive interim data from the first part of the ongoing phase II study. This part of the study met its two primary endpoints of demonstrating safety and tolerability of the combination regimen and the effect of the VX-770 and VX-809 combination on CFTR function as measured by sweat chloride, which is a key measure of the CFTR protein function.

Vertex Pharma plans to initiate the second part of the study in the fourth quarter of 2011, after it completes further analyses of the first part data.

Besides further evaluating the effect of the combined multiple doses of VX-770 and VX-809 on sweat chloride measurements, the second part of the study would also assess longer dosing periods for the combination drug.

The CFTR protein does not reach the cell surface in normal amounts in patients with the F508del mutation. This leads to an increase in the amount of mucus and other complications that can damage the lung. We note that VX-809 helps the protein reach the cell surface, while Kalydeco helps the protein function normally once it reaches the cell surface.

We note that on approval, Kalydeco might face competition from only one other drug – ataluren. Ataluren, which is currently in late-stage development, is being developed by PTC Therapeutics Inc. as a treatment for CF. PTC Therapeutics is developing ataluren in collaboration with Sanofi-Aventis (SNY).

Neutral on Vertex Pharma

We currently have a Neutral recommendation on Vertex Pharma. The stock carries a Zacks #3 Rank (Hold rating) in the short-run. We are encouraged by the NDA filing of Kalydeco, as the company presently markets only one drug, Incivek, for the treatment of chronic hepatitis C.