FDA triples number of CF mutations that Kalydeco can treat

18 May 2017

By expanding the approved use of Kalydeco (ivacaftor) in cystic fibrosis (CF), the US Food and Drug Administration (FDA) has tripled the number of rare gene mutations that the drug can now treat.

The indication of the Vertex Pharmaceuticals (Nasdaq: VRTX) product has been expanded from the treatment of 10 mutations, to 33, with the FDA basing its decision, in part, on the results of laboratory testing, which it used in conjunction with evidence from earlier trials.

"We will continue to work closely with the FDA to bring Kalydeco to more people with responsive mutations as rapidly as possible

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