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FDA Advisors Recommend Approval of Kalydeco (Ivacaftor) for Use in Cystic Fibrosis Patients Aged 6 and Older With R117H Mutation

Approval decision set for December

The FDA’s Pulmonary Allergy Drugs Advisory Committee (PADAC) has voted 13–2 to recommend approval of Kalydeco (ivacaftor, Vertex Pharmaceuticals) in cystic fibrosis (CF) patients 6 years of age and older who have the R117H mutation in the cystic fibrosis transmembrane regulatory (CFTR) gene, which is the indication currently being reviewed by the FDA.

Advisory committees provide the FDA with independent scientific and medical advice on the safety, effectiveness, and appropriate use of potential new medications. The FDA is not bound by the committee’s recommendations but often follows its advice.

The FDA is expected to make a decision on the approval of ivacaftor by December 30, 2014 under the Prescription Drug User Fee Act (PDUFA).

CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Kalydeco (ivacaftor) is currently approved to treat more than 2,600 patients aged 6 years and older in North America, Europe, and Australia who have specific mutations in the CFTR gene. In the U.S., these mutations include G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, and G1349D.

In patients with the R117H mutation, the CFTR protein reaches the cell surface but does not function properly. In the U.S., approximately 500 patients 6 years of age and older have this mutation.

Ivacaftor is a CFTR protein potentiator indicated for the treatment of CF in patients aged 6 years and older who have one of the following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R. The CFTR protein is a chloride channel present on the surface of epithelial cells in multiple organs. Ivacaftor facilitates increased chloride transport by potentiating the “channel open” probability (or gating) of the CFTR protein.

Ivacaftor is not effective in patients with CF who are homozygous for the F508del mutation in the CFTR gene.

Sources: Vertex Pharmaceuticals; October 21, 2014; and Kalydeco Prescribing Information; June 2014.

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