4:55 PM
Feb 12, 2018
 |  BC Extra  |  Company News

Third CF candidate from Vertex approved

Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) said FDA approved Symdeko tezacaftor/ivacaftor to treat cystic fibrosis in patients ages 12 and older who have two copies of the delta F508 mutation or who have at least one CF gene mutation that is responsive to Symdeko. The company said it will begin shipping the product to U.S. pharmacies this week. Symdeko has an annual wholesale acquisition cost of $22,400 for a 28-day pack.

An MAA for Symdeko is under EMA review with a decision expected next half.

Vertex also markets Kalydeco ivacaftor and Orkambi ivacaftor/lumacaftor for multiple CF indications. A 28-day supply of Kalydeco has a WAC of $23,896.13, and the same supply of Orkambi has a WAC of $20,919.23.

Kalydeco is a small molecule potentiator of cystic fibrosis transmembrane conductance regulator (CFTR), and Orkambi is a fixed-dose combination of Kalydeco and lumacaftor (VX-809), a small molecule CFTR corrector.

Symdeko is a fixed-dose tablet combination of tezacaftor, a small molecule corrector of CFTR, and ivacaftor.

Ivacaftor and tezacaftor are being developed under an extended 1998 deal with the not-for-profit Cystic Fibrosis Foundation’s Cystic Fibrosis Foundation Therapeutics (CFFT) drug development affiliate to discover therapeutics to treat CF. In 2014, Royalty Pharma (New York, N.Y.) purchased from the foundation the royalty rights for CF treatments developed by Vertex, including ivacaftor and tezacaftor.

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