Vertex' Kalydeco Approved for an Additional Indication - Analyst Blog
February 24 2014 - 1:00PM
Zacks
Vertex Pharmaceuticals
Inc. (VRTX) received encouraging news when the U.S. Food
and Drug Administration (FDA) approved Kalydeco for an additional
indication. Kalydeco was approved for treating patients (age ≥ 6
years) suffering from cystic fibrosis (CF) who have one of eight
additional mutations in the cystic fibrosis transmembrane
conductance regulator (CFTR) gene.
Kalydeco is currently marketed for treating CF patients (age ≥ 6
years) who have at least one copy of the G551D mutation. Following
the approval for the new indication, Kalydeco can be used to treat
CF patients with the following nine mutations: G551D, G178R, S549N,
S549R, G551S, G1244E, S1251N, S1255P and G1349D.
Kalydeco’s approval for the new indication was based on phase III
data that demonstrated statistically significant improvements in
lung function in patients on Kalydeco monotherapy. The safety
profile was similar to that seen in earlier phase III studies.
Approval in the U.S. for the new indication was sought in Oct 2013.
Similar submissions for Kalydeco were also made in Europe, Canada
and Australia for the same indication.
Vertex Pharma is also evaluating Kalydeco in children with CF in
the age group of 2–5 years 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. The company intends to
seek U.S. approval in this indication in the second half of 2014.
However, in December last year, Vertex Pharma 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 endpoint. The company is also evaluating
Kalydeco for other indications.
Meanwhile, Vertex Pharma has been witnessing encouraging Kalydeco
sales since its launch. The company reported Kalydeco sales of
$371.3 million in the year 2013, an increase of 116.4% from the
year-ago period. Kalydeco sales are expected in the range of $470
million to $500 million in 2014.
Vertex Pharma maintained its total revenue guidance for 2014 in the
range of $570 million to $600 million. The Zacks Consensus Estimate
of $603 million is slightly above the company’s guidance range.
Vertex Pharma carries a Zacks Rank #3 (Hold). Some better-ranked
stocks include Alexion Pharmaceuticals, Inc.
(ALXN), Actelion Ltd. (ALIOF) and
Alkermes (ALKS). While Alexion holds a Zacks Rank
#1 (Strong Buy), Actelion and Alkermes carry a Zacks Rank #2
(Buy).
ACTELION LTD (ALIOF): Get Free Report
ALKERMES INC (ALKS): Free Stock Analysis Report
ALEXION PHARMA (ALXN): Free Stock Analysis Report
VERTEX PHARM (VRTX): Free Stock Analysis Report
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