Vertex Pharmaceuticals (MA) Surges As FDA Advisory Panel Approves Kalydeco's Label Expansion

Published: Oct 22, 2014

Vertex Pharmaceuticals Surges As FDA Advisory Panel Approves Kalydeco's Label Expansion

October 22, 2014

By Jessica Wilson, BioSpace.com Breaking News Staff

Vertex Pharmaceuticals Incorporated announced on Tuesday that the U.S. Food and Drug Administration Pulmonary Allergy Drugs Advisory Committee (PADAC) voted to recommend approval of the company’s cystic fibrosis drug, Kalydeco, for a new group of patients.

Shares if Vertex rose 3.5 percent on the news.

The FDA will vote on the approval by the end of 2014. Though the FDA is not bound by the recommendations of the PADAC, the agency usually follows the advice of the advisory committee.

The FDA approved Kalydeco, the first medicine to treat the underlying cause of CF in people with specific mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, in 2012. According to the Boston Business Journal,Kalydeco earned $371 million in revenue for Vertex in 2013.

The recommended approval would be for treatment in patients with another genetic mutation associated with the disease, called R117H. The Wall Street Journal reports, “This mutation…accounts for about 750 cystic fibrosis patients, or 2.7% of the U.S. patient population, and could add as much as $160 million in additional revenue by 2017.”

“Today’s recommendation is a positive and important step toward making ivacaftor available for people ages 6 and older with the R117H mutation,” said Jeffrey Chodakewitz, senior vice president and chief medical officer at Boston-based biotechnology Vertex in a press release.

The recommended approval was not certain and is surprising given that the drug failed to meet its primary endpoint in the study, except for in a group of patients 18 years and older. In that population, patients experienced statistically significant approval in lung function. Perhaps the committee took into consideration that patients aged 12 to 17 years could not be assessed because no one of that age was enrolled in the study.

Though the approval of Kalydeco for an additional 750 patients is important, analysts have been looking to the result as a ‘soft indicator’ for the future of the Kalydeco program at Vertex. Next year, Vertex plans to submit for FDA approval a treatment that combines Kalydeco and another medicine, a combination that has already demonstrated statistical significance in clinical trials.

“A firm endorsement by the advisory committee and the agency would suggest a generally positive view of the program, boding well for approval of the much more important combination next year with a broad and positive label,” wrote Sanford Bernstein analyst Geoff Porges in an investor note.

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