Vertex Pharmaceuticals Inc.
) recently received European approval of Kalydeco for treating
people suffering from cystic fibrosis (CF). The European Commission
approved the drug to treat patients with CF, aged 6 years and above
and who have at least one copy of the G551D mutation in the cystic
fibrosis transmembrane conductance regulator (CFTR) gene. Vertex
intends to launch Kalydeco in Europe shortly.
The European approval did not come as a surprise as in May this
year, the European Committee for Medicinal Products for Human Use
(CHMP) voted in favor of approving the drug. Kalydeco is the first
medicine that targets the underlying cause of CF, which is a rare
genetic disease, caused by defective or missing CFTR proteins.
Approximately 1,100 people have this mutation in Europe.
Notably, Vertex conducted two global phase III studies,
determining the efficiency of Kalydeco in treating patients with
CF. These studies revealed that Kalydeco improves breathing and
other symptoms of CF patients aged 6 years and above.
Patients on Kalydeco showed improvements in lung function,
weight gain and certain quality of life measurements in comparison
to those on placebo. Favorable results from these studies backed
the CHMP's positive opinion.
Vertex has a collaboration agreement with Cystic Fibrosis
Foundation Therapeutics, Inc. for Kalydeco. According to the
agreement, Cystic Fibrosis is entitled to receive royalties on net
sales of Kalydeco.
Kalydeco is already available in the US, where it was approved
in January 2012 for treating patients suffering from CF, with at
least one copy of the G551D mutation in the CFTR gene. Vertex
recorded sales of $18.4 million for the drug in the first quarter
Currently, Vertex has another product marketed in the EU:
Incivo. Vertex has an agreement with
Johnson & Johnson
) to commercialize Incivo outside the US.
Given the strong first-quarter sales of Kalydeco in the US along
with the EU approval, we believe the drug can be a major growth
driver in the future. We expect investor focus to remain on
Kalydeco's performance in the US as well as its launch progress in
We currently have a Neutral recommendation on Vertex. The stock
carries a Zacks #3 Rank (Hold rating) in the short run.
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