Vertex Pharmaceuticals (MA) Submits Supplemental New Drug Application (sNDA) To FDA For Use Of KALYDECO® (Ivacaftor) In People 18 And Older With Cystic Fibrosis Who Have The R117H Mutation
Published: Jul 01, 2014
BOSTON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the submission of a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for the approval of KALYDECO® in people with cystic fibrosis (CF) ages 18 and older who have the R117H mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. In the United States, KALYDECO is currently approved for use in people with CF ages 6 and older who have one of the following nine mutations: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P or G1349D. CF is caused by a defective or missing CFTR protein that results from mutations in the CFTR gene. In the United States, approximately 300 people have the R117H mutation and are 18 years of age or older. R117H is the most common residual function mutation and also has a defect in the gating of the CFTR protein.
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