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FDA expands approval of Kalydeco to include additional cystic fibrosis mutations
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The FDA has extended approval for ivacaftor to treat 23 additional mutations of cystic fibrosis among pediatric patients aged older than 2 years.
Previously, ivacaftor (Kalydeco, Vertex) was only FDA-approved to treat patients with one of 10 mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. With approval extended to 33 mutations, an additional 3% of patients with cystic fibrosis will now be eligible to receive ivacaftor in the United States.
“Many rare cystic fibrosis mutations have such small patient populations that clinical trial studies are not feasible,” Janet Woodcock, MD, director of the FDA’s Center for Drug Evaluation and Research, said in a press release. “This challenge led us to using an alternative approach based on precision medicine, which made it possible to identify certain gene mutations that are likely to respond to Kalydeco.”
The FDA based its approval, in part, on the results of laboratory testing, as well as evidence from earlier clinical trials, a unique approach which provides a pathway for adding additional, rare mutations of the disease, based largely on in vitro data.
“We are encouraged by the FDA’s willingness to explore innovative ways to make highly effective medicines like Kalydeco with a well-established safety profile available to more people who are in urgent need,” Jeffrey Chodakewitz, MD, executive vice president and chief medical officer at Vertex, said in a press release. “We will continue to work closely with the FDA to bring Kalydeco to more people with responsive mutations who are still in need as rapidly as possible.”
In place of clinical trial data, the FDA noted that an in vitro cell-based model system has been shown to “reasonably predict clinical response” to ivacaftor. The 23 additional mutations demonstrated a clinical relevant response to ivacaftor during laboratory testing which indicated that patients with these mutations could obtain clinically meaningful benefit.
“Cystic fibrosis treatment has advanced rapidly, but there is need for broader access to these important medicines and development of additional medicines remains urgent,” Patrick Flume, MD, director of the Medical University of South Carolina Cystic Fibrosis Center, said in the release. “The use of in vitro data to support this approval is an important step forward in making medicines like Kalydeco available to more patients, especially those with rare mutations.”