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FDA Panel Backs Vertex's Cystic Fibrosis Drug Orkambi

Vertex Pharmaceuticals Inc. (VRTX) said Tuesday that the U.S. Food and Drug Administration's Pulmonary-Allergy Drugs Advisory Committee has voted 12 to 1 to recommend the approval of Orkambi for use in people with cystic fibrosis ages 12 and older who have two copies of the F508del mutation in the CFTR gene.

The FDA is expected to make a decision on the approval of Orkambi by July 5 under the Prescription Drug User Fee Act. The FDA is not bound by the committee's recommendation but often follows its advice.

If approved, Orkambi will be the first and only medicine to treat the underlying cause of cystic fibrosis for eligible people with cystic fibrosis ages 12 and older with two copies of the F508del mutation in the CFTR gene.

People with two copies of the F508del mutation represent the largest group of people with cystic fibrosis. There are about 8,500 people ages 12 and older with two copies of the F508del mutation in the U.S.

Cystic fibrosis is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia. Children must inherit two defective CFTR genes — one from each parent — to have cystic fibrosis.

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