Vertex Pharmaceuticals Inc.
) gained 1.24% on the submission of a supplemental new drug
application (sNDA) to the FDA for Kalydeco (ivacaftor) for the
treatment of cystic fibrosis (CF) in patients (aged ≥18 years) with
the R117H mutation in the cystic fibrosis transmembrane conductance
regulator (CFTR) gene.
Vertex Pharma filed the sNDA based on phase III study results. We
note that although Kalydeco had not met the primary endpoint of
mean absolute change from baseline compared to placebo in a phase
III study in CF patients aged ≥6 years, a pre-specified 24-week
long subset analysis in 50 patients aged ≥18 years showed
statistically significant improvements in lung function apart from
other key secondary endpoints.
Vertex Pharma will file the marketing application for the R117H
mutation in the third quarter of 2014 in Europe. Last week,
Kalydeco received a positive opinion from the European Committee
for Medicinal Products for Human Use (CHMP) for CF in patients aged
≥6 years, who suffer from one of eight non-G551D gating mutations
in the CFTR gene. A response in the EU regarding final approval
should be out in the September-October timeframe.
Kalydeco is already approved in the U.S. for the treatment of CF in
patients aged ≥6 years, suffering from one of nine mutations
including G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P
or G1349D. In Europe, Kalydeco is approved for CF in patients (aged
≥6 years) suffering from at least one type of the G551D mutation.
We are positive on Vertex Pharma's label expansion efforts for
Kalydeco. In 2013, Kalydeco sales were $371.3 million, up 116.4%
y/y. Kalydeco's approval in the U.S. for the R117H mutation will
provide access to nearly 300 eligible patients.
Vertex Pharma carries a Zacks Rank #3 (Hold). Some better-ranked
stocks in the health care sector include
Biogen Idec Inc.
ANI Pharmaceuticals, Inc.
). All these stocks hold a Zacks Rank #1 (Strong Buy).
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