Updates on the regulatory front are eagerly awaited by investors
in the pharma/biotech sector as they impact the share price of the
concerned company. Regulatory updates include events like filing of
marketing applications, acceptance of these applications for review
by the regulatory agencies, reviews by advisory panels and finally,
a response from the regulatory agency regarding the approval
Late last week,
Vertex Pharmaceuticals Inc.
) cystic fibrosis (CF) drug, Kalydeco, received approval in the EU
for eight additional gating mutations. Kalydeco, which was
initially approved for the treatment of CF patients (aged ≥6 years)
who have at least one copy of the G551D mutation, can now be used
in patients (aged ≥6 years) who have one of eight non-G551D gating
mutations (G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and
G1349D) in the cystic fibrosis transmembrane conductance regulator
The European Commission's (EC) decision is not surprising since the
Committee for Medicinal Products for Human Use (CHMP) had earlier
recommended the approval of Kalydeco in this indication.
We note that Kalydeco gained approval for these additional mutation
in Feb 2014 in the U.S. Vertex Pharma is currently seeking FDA
approval for Kalydeco for the treatment of CF patients (aged ≥18
years) with the R117H mutation.
Moreover, Vertex Pharma is developing a combination of Kalydeco and
lumacaftor for patients aged 12 years and more who are suffering
from two types of the F508del mutation. The company intends to file
a new drug application for this combination on a rolling basis.
Vertex Pharma expects to complete the submission in the fourth
quarter of 2014.
We are encouraged by Vertex Pharma's continued progress with its CF
franchise. The company is going through a transition period given
its decision to focus on its CF franchise and drop investment in
the hepatitis C virus pipeline. Kalydeco's approval in the EU for
the additional gating mutations will provide access to nearly 250
In the first half of 2014, Kalydeco revenues were $212.6
million. This approval will help the company achieve its targeted
Kalydeco revenues of $470 million − $500 million in 2014.
Vertex Pharma carries a Zacks Rank #3 (Hold). Some better-ranked
stocks in the health care sector are Actelion (
), Salix Pharmaceuticals Ltd. (
) and Endo International plc (
). All these stocks hold a Zacks Rank #1 (Strong Buy).
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