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FDA panel recommends Vertex cystic fi...
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FDA panel recommends Vertex cystic fibrosis treatment
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WASHINGTON: A U.S. Food and Drug Administration advisory committee on Tuesday recommended approval of Vertex Pharmaceuticals Inc’s combination cystic fibrosis therapy, finding it to be safe and effective for patients ages 12 and up.

The recommendation, which came in a 12-1 vote, will now be considered by FDA officials who are expected to decide by July 5 whether to approve the treatment, Orkambi, for sale in the United States. The FDA usually follows the recommendations of its advisory panels but does not have to.

“Is the drug going to be approved? Yes,” said RBC Capital Markets analyst Michael Yee, who forecast annual peak Orkambi sales of up to US$5 billion.

Yee said the panel vote removed uncertainty that had been holding back the Vertex share price, and that Orkambi would return the company to profitability.

The Vertex stock had been halted during the panel meeting, but when trading resumed shares jumped 7 percent to US$132.80.

Cystic fibrosis is a life-shortening lung disease that afflicts about 30,000 people in the United States.

Orkambi combines the FDA-approved Vertex drug Kalydeco and a new compound called lumacaftor. The treatment targets the most common genetic mutation responsible for cystic fibrosis.

Vertex estimates that Orkambi would initially be used to treat about 8,500 patients with the F508del mutation, or 28 percent of U.S. cystic fibrosis sufferers. Kalydeco, the first medicine to address the underlying cause of CF rather than just symptoms, is currently used to treat about 2,000 patients who suffer from a different mutation.

Worldwide, there are an estimated 70,000 CF patients, about half of whom have the F508del mutation Orkambi is designed to treat.

Those patients who were not eligible for Kalydeco are currently treated with a cocktail of vitamins, pain killers and antibiotics to maintain daily functions.

Before reaching its final decision, a divided panel was unable to say that lumacaftor had a positive effect when used in combination with Kalydeco because the data was insufficient to demonstrate the efficacy of Orkambi’s separate components.

Agency staff offered a mixed review of the combination last week, raising questions about whether it had an added benefit over Kalydeco alone.

The committee also heard compelling and often tearful public testimony from nearly two dozen CF patients, their relatives and advocates, who pleaded that it recommend Orkambi on the hope the treatment could slow or even reverse progression of the disease for some.

(Additional reporting by Bill Berkrot in New York; Editing by Diane Craft and Ted Botha)

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