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 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
$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 $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.
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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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