FDA Expands Approved Use of Ivacaftor for Cystic Fibrosis

The US Food and Drug Administration (FDA) has granted expanded approval to ivacaftor (Kalydeco®, Vertex Pharmaceuticals), according to an agency press release.¹

Previously, ivacaftor was indicated to treat 10 rare gene mutations associated with cystic fibrosis; the FDA approval increases that number to 33. The expansion follows positive laboratory data and the results from previous human clinical trials.

Available as tablets or oral granules, ivacaftor allows proteins created by the cystic fibrosis transmembrane conductance regulator (CFTR) gene to function, improving lung function and weight gain. Ivacaftor is approved for use in patients age 2 and older who have 1 CFTR gene mutation that is — based on laboratory or clinical data — responsive to pharmaceutical treatment.

“Many rare cystic fibrosis mutations have such small patient populations that clinical trial studies are not feasible,” noted Janet Woodcock, MD, director of the FDA Center for Drug Evaluation and Research in Silver Spring, Maryland, in the 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 [ivacaftor].”

Side effects include headache, upper respiratory tract infection, nasal or sinus congestion, abdominal pain, diarrhea, rash, nausea, and dizziness. Ivacaftor is also associated with risks including elevated transaminases and pediatric cataracts.

Reference

FDA expands approved use of Kalydeco to treat additional mutations of cystic fibrosis [news release]. Silver Springs, MD: US Food and Drug Administration. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm559212.htm. Published May 17, 2017. Accessed May 18, 2017.