Vertex Pharmaceuticals Incorporated (VRTX) has been showing progress in its efforts to expand the label of its cystic fibrosis (CF) drug, Kalydeco. Kalydeco, which is currently approved for use in CF patients (age ≥ 6 years) who have at least one copy of the G551D mutation, is a key product in Vertex Pharma’s portfolio.
Kalydeco, launched in 2012, is off to a strong start with first half 2013 sales increasing 151% to $160.8 million. The impressive performance should continue with the company working on expanding the label. Vertex Pharma is currently looking to get Kalydeco monotherapy approved for CF patients (age ≥ 6 years) who have at least one non-G551D gating mutation in the cystic fibrosis transmembrane conductance regulator (:CFTR) gene.
The supplemental new drug application (sNDA) for this indication was submitted to the FDA recently and was based on phase III data that demonstrated statistically significant improvements in lung function in patients on Kalydeco monotherapy. The safety and tolerability profile was similar to that seen in earlier phase III studies. Long-term data was also submitted from the 96-week PERSIST open-label study.
Vertex Pharma intends to seek EU approval for this indication as well with a Marketing Authorization Application (MAA) variation slated to be submitted in October.
Meanwhile, Vertex Pharma is evaluating Kalydeco in combination with VX-809 – a new drug application for this combination could be submitted next year. Kalydeco is also being studied in combination with VX-661.
Vertex Pharma currently carries a Zacks Rank #4 (Sell). At present, companies like Actelion Ltd. (ALIOF), Seattle Genetics Inc. (SGEN) and Alexion Pharmaceuticals (ALXN) look more attractive. While Actelion and Seattle Genetics are Zacks Rank #1 (Strong Buy) stocks, Alexion is a Zacks Rank #2 (Buy) stock.