ARTICLE | Company News

FDA approves Kalydeco for more CF mutations

February 22, 2014 1:46 AM UTC

FDA approved an sNDA from Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) expanding the label for cystic fibrosis drug Kalydeco ivacaftor to include treatment of eight additional mutations in the CF transmembrane conductance regulatory ( CFTR) gene. Kalydeco is already approved in the U.S. to treat CF patients ages six years and older who have at least one copy of the G551D mutation in the CFTR gene. The drug is now also approved for CF patients with at least one copy of the following mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D.

Vertex said about 150 patients in the U.S. have one of the additional mutations. The company did not change its 2014 Kalydeco revenue outlook of $470-$500 million. Kalydeco revenues in 2013 were $371.3 million. ...