Vertex Pharmaceuticals Inc.'s experimental cystic fibrosis
treatment Orkambi won the backing of a Food and Drug Administration
advisory committee on Tuesday, which voted 12-1 to recommend
approval.
The FDA is expected to make a final decision on whether to
approve Orkambi by July 5. The FDA isn't required to follow the
committee's recommendation, but it usually does. The FDA advisory
committee comprises 13 independent doctors, scientists, and
consumer and patient representatives.
U.S. approval of Orkambi is seen by analysts as essential to
Vertex's success in the coming years. The Boston, Mass., company
was founded in 1989, and had lost an accumulated $4.9 billion as of
March 31. Vertex, whose shares were halted during regular trading
on Tuesday, has a market value of more than $30 billion. If
approved, Orkambi could reach global sales of $1.6 billion next
year and $4.2 billion in 2020, according to J.P. Morgan.
In clinical studies, the lung function of patients taking
Orkambi improved by a range of 2.6 percentage points to 3
percentage points compared with that of patients receiving placebo.
FDA officials called the drug's effect "modest," but said it was a
statistically significant improvement compared with the
lung-function declines experienced by patients receiving
placebos.
Write to Joseph Walker at joseph.walker@wsj.com
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