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Kalydeco: New Drug Helps Some with Cystic Fibrosis
On January 31, the FDA approved ivacaftor (Kalydeco), the first-ever drug that treats an underlying cause of cystic fibrosis. In about 4% of cystic fibrosis patients, defective chloride ion channel transporters (CFTRs) are trafficked to the cell membrane, but don't work properly. Kalydeco "unlocks" and improves function of CFTR, improving lung function and quality of life. The benefits were established in 2 randomized trials of 231 patients total, in which those taking Kalydeco had a mean absolute improvement in FEV1 of ~11% predicted, sustained through one year.
About 1,200 people living with cystic fibrosis in the U.S. have the particular mutation (G551D in the CFTR gene) that would allow them to benefit from ivacaftor.
Most people with CF have the delta 508 mutation, in which defective CFTR ion channels never make it to the cell membrane. Kalydeco would not be expected to help them, but the drug is being tested in combination with other agents in clinical trials among this much larger population.
Vertex Pharmaceuticals produced Kalydeco (a.k.a. VX-770) assisted by $75 million in research funding from the Cystic Fibrosis Foundation, the leading CF patient advocacy organization. The Cystic Fibrosis Foundation will share in royalties from the sale of Kalydeco: a novel financial relationship in the highly interconnected world of industry, academia, and patient advocacy organizations. The CF Foundation told media it would reinvest its royalties into further cystic fibrosis research.
How much will ivacaftor/Kalydeco cost?
$294,000 a year (per patient)
Sales are expected to be in the hundreds of millions of dollars per year. Vertex saw their stock rise 6% on the news of FDA approval, to $37.