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Pharmaceuticals

Vertex Cystic Fibrosis Drug Gets Green Light

by Lisa M. Jarvis
February 6, 2012 | A version of this story appeared in Volume 90, Issue 6

Vertex Pharmaceuticals has received FDA approval for Kalydeco, the first drug to treat the underlying cause of cystic fibrosis rather than just the symptoms. The regulatory green light came nearly three months ahead of FDA’s deadline for making a decision. Kalydeco, which increases the flow of water and salt across cell membranes, is approved to treat only the small subset of the cystic fibrosis population carrying a genetic mutation called G551D. Vertex plans to charge $294,000 annually for the pill, which is taken twice daily.

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