Thursday, October 20, 2011

VX-770 submitted to FDA for approval

Vertex, the pharmaceutical research firm that has developed VX-770 (now called KALYDECO) has started the FDA process to have this drug approved for CF patients with the G551D mutation. Although Peter does not have this mutation (only about 4 percent of CFers do), we are still thrilled for VX-770 to be headed towards the market. You can read the CF Foundation's press release here

KALYDECO/VX-770 is the first drug to address the root cause of cystic fibrosis. In CF Foundation CEO Robert Beale's words, it is "proof of concept" that a drug can change how cells interact with the faulty CFTR protein. You can read my post on how VX-770 works here

Of course, we are most excited about VX-809 in combination with VX-770 which may make a difference for the majority of patients with CF. In any case, we are thrilled to have research going forward to extend the life of our little boy and other CF patients. Yay, Vertex!


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