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FDA panel recommends approval for Vertex combination, despite efficacy issues

WASHINGTON – Federal health advisers are recommending approval for an experimental combination drug to treat cystic fibrosis, despite unresolved questions about the impact of one of the pill’s ingredients.

Vertex Pharmaceuticals has asked the Food and Drug Administration to approve its twice-a-day drug for patients with the most common form of cystic fibrosis, a deadly inherited disease that causes sticky mucus buildup in the lungs.

A panel of FDA expert advisers voted 12-1 in favour of approving the drug, based on safety and effectiveness data submitted by Vertex.

Vertex’s new drug, which would be sold under the brand name Orkambi, is the company’s follow-up to its breakthrough pill Kalydeco, which became the first drug to treat the underlying cause of cystic fibrosis in 2012. Orkambi combines Kalydeco with a new drug, lumacaftor.