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FDA approves Kalydeco for patients with specific cystic fibrosis mutation
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Vertex Pharmaceuticals has received FDA approval for ivacaftor to treat cystic fibrosis among patients, aged 6 years and older, with the R117H mutation.
The approval follows a recommendation from the FDA’s Pulmonary-Allergy Drugs Advisory Committee to approve ivacaftor (Kalydeco, Vertex) in patients with the mutation.
Data from a phase 3 study of ivacaftor, which included 69 patients, aged 6 years and older, with cystic fibrosis and the R117H mutation, served as the basis for the approval, according to a company press release.
“[This] approval marks an important milestone for people with the R117H mutation who will now have a medicine to treat the underlying cause of their disease for the first time,” Jeffrey Chodakewitz, MD, executive vice president and chief medical officer at Vertex, said in the release.
Kalydeco now is approved for use among patients aged 6 years and older with cystic fibrosis with the R117H, G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P or G1349D mutation.
Perspective
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Edward A. Bell, PharmD, BCPS
With this announcement of efficacy for an additional genetic mutation, the R117H mutation, ivacaftor (Kalydeco, Vertex) is now labeled by the FDA for use in individuals 6 years of age and older with cystic fibrosis (CF) with any of 10 specific mutations. Kalydeco was initially labeled by the FDA for use in CF in 2012 for the G551D mutation.
These additional uses significantly broaden the number of children and adults with CF that can benefit from ivacaftor’s unique mechanism of action — the ability to interact with the underlying cause of CF, the cystic fibrosis transmembrane conductance regulator (CFTR) protein. With the R117H mutation, CFTR is transported to the cell membrane, but does not function properly.
Dramatic improvements in survival have occurred in individuals with CF in recent decades, in large part due to new and improved pharmacotherapies. The median predicted age of survival is now in the early 40 years. Hope and optimism for individuals with CF and their families are dramatically increasing.
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