FDA Approves Ivacaftor for People with R117H Mutation of Cystic Fibrosis

The U.S. Food and Drug Administration (FDA) announced today that it has approved the use of ivacaftor (Kalydeco™) to treat people with cystic fibrosis ages 6 and older who have the R117H mutation.

Dec. 29, 2014 | 2 min read

Developed by Vertex Pharmaceuticals Inc. with significant clinical and financial support from the CF Foundation, the drug is the first to target the underlying cause of the disease - a defective CFTR protein caused by mutations in the CF gene.

About 500 people with CF in the United States ages 6 and older have the R117H mutation.

Following today's announcement, the therapy is now available to people with CF ages 6 and older who have at least one of 10 rare mutations of CF. Ivacaftor was initially approved in 2012 for people ages 6 and older with the G551D mutation.

“The FDA's decision is another great step forward in our efforts to treat the basic genetic defect in more people with CF,” said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. “In addition to supporting research of ivacaftor in other patient groups - as a single therapy and in combination with other potential drugs - we are pursuing exciting new opportunities to find lifesaving treatments for all people living with this disease.”

In people with the R117H mutation, the CFTR protein reaches the cell surface but does not function normally, interfering with the proper flow of salt and water in and out of the cell. Ivacaftor helps restore the function of the faulty protein.

The FDA's expanded approval is based on results from a Phase 3 trial of the drug in people ages 6 and older with the R117H mutation and follows a recommendation in October 2014 by an independent FDA advisory committee to approve ivacaftor for this patient group.

Ivacaftor was previously approved for people with CF ages 6 and older with at least one of the following mutations: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D.

The FDA is currently reviewing a new drug application for ivacaftor in children ages 2 to 5 with these mutations, with a decision expected in 2015.

Read Vertex's press release.

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