US FDA approves wider use for Vertex’ Kalydeco

US biotech firm Vertex Pharmaceuticals (Nasdaq: VRTX) says the US Food and Drug Administration has approved a supplemental New Drug Application for the use of Kalydeco (ivacaftor) in people with cystic fibrosis (CF) aged six and older who have the R117H mutation in the cystic fibrosis transmembrane conductance regulator (CFTR)gene. Vertex shares gained 1.2% to $119.79 following the announcement on Monday.

This approval follows a recommendation by the FDA’s Pulmonary-Allergy Drugs Advisory Committee (PADAC) to approve the medicine in this group of people with CF, by a 13 to two vote. Kalydeco is now approved for use in the USA in people aged six and older with CF with one of the following 10 mutations: R117H, G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P or G1349D. The approval is based on previously announced data from a Phase III study of ivacaftor that enrolled 69 people with CF aged six and older who had the R117H mutation.

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