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Vertex Gets Quick FDA Approval Of Kalydeco; Prices New CF Drug At $294,000 Per Year

Kalydeco, an oral CFTR potentiator, becomes the first approved drug to address the underlying cause of cystic fibrosis, rather than just treating the disease’s symptoms.

The targeted nature and ultra-orphan setting for Vertex Pharmaceuticals Inc.’s cystic fibrosis medication Kalydeco means that the company will be able to launch the groundbreaking therapy, its second commercial product, with a small force and still achieve significant success on the market.

FDA announced the approval of Kalydeco (ivacaftor, VX-770) for a subpopulation of cystic fibrosis patients on Jan. 31, more than two months ahead of the drug’s PDUFA date

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