Vertex Pharmaceuticals Incorporated
) 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
Seattle Genetics Inc.
) look more attractive. While Actelion and Seattle Genetics are
Zacks Rank #1 (Strong Buy) stocks, Alexion is a Zacks Rank #2
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