Vertex Pharmaceuticals Inc.
) recently suffered a setback when its phase III study on
Kalydeco for the indication of cystic fibrosis (CF) in patients
(age ≥ 6 years) with R117H mutation, failed to meet its primary
We note that Vertex Pharma is seeking to expand the label of
its CF drug, Kalydeco. Kalydeco is currently approved for use in
CF patients (age ≥ 6 years) who have at least one copy of the
The randomized phase III study (n=69) spanned 24 weeks and
evaluated the efficacy of Kalydeco in patients who have at least
one R117H mutation. The study failed to meet its primary endpoint
which was the absolute change from baseline in FEV1 (percent
predicted forced expiratory volume in one second) throughout the
treatment period of Kalydeco as compared to placebo across all
However, Vertex Pharma believes that the results show clinical
benefits for patients aging 18 and above. Hence, the company
plans to meet with the U.S. Food and Drug Administration (FDA) in
early 2014 to discuss the phase III study data and subsequent
potential submission of a supplemental New Drug Application
(sNDA) for patients with R117H mutation.
We remind investors that Vertex Pharma has two marketed
products - Kalydeco and Incivek (for HCV infection).
Kalydeco is a key product in Vertex Pharma's portfolio and the
company is keen to expand its label for additional
Vertex Pharma is also 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.
Concurrent with the phase III study results, Vertex Pharma
announced that the FDA has accepted its sNDA for Kalydeco for
gating mutations study and granted a priority review status to
the application. Under the Prescription Drug User Fee Act
(PDUFA), a decision by the FDA is expected by Mar 27, 2014.
Meanwhile, Vertex Pharma is also evaluating Kalydeco in
children with CF in the age group of 2-5 who have a gating
mutation. The study is fully enrolled and currently ongoing. The
company expects data from the study in the second quarter of 2014
and a potential NDA submission in the second half of 2014.
On the other hand, enrollment is complete in a phase II
proof-of-concept study evaluating Kalydeco in patients (age ≥ 12
years) with CF who have clinical evidence of residual CFTR
function. Vertex Pharma expects data from this study in the
second quarter of 2014.
Vertex Pharma currently carries a Zacks Rank #3 (Hold). Right
now, stocks that look attractive include
). While Actelion carries a Zacks Rank #1 (Strong Buy),
Affymetrix and Amgen are Zacks Rank #2 (Buy) stocks.
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