FDA approves supplemental new drug application for the use of Kalydeco

BOSTON — Vertex Pharmaceuticals has announced that the Food and Drug Administration approved a supplemental new drug application for the use of Kalydeco (ivacaftor) in people with cystic fibrosis ages 6 years and older who have the R117H mutation in the cystic fibrosis transmembrane conductance regulator gene.



The approval follows a recommendation by the FDA’s Pulmonary-Allergy Drugs Advisory Committee to approve the medicine in this group of people with CF. Kalydeco is now approved for use in the United States in people ages 6 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 3 study of ivacaftor that enrolled 69 people with CF ages 6 years and older who had the R117H mutation.



Cystic fibrosis is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. In people with the R117H mutation, the CFTR protein reaches the cell surface but does not function properly. Approximately 500 people with CF ages 6 and older have this mutation in the United States. With the approval, Kalydeco is now approved to treat more than 3,100 people ages 6 and older in North America, Europe and Australia who have specific mutations in the cystic fibrosis transmembrane conductance regulator gene.



In July, Vertex submitted a Marketing Authorization Application variation to the European Medicines Agency for the approval of Kalydeco for people with the R117H mutation.