FDA Expands Approval of Cystic Fibrosis Drug

The US Food and Drug Administration has expanded the approval of Kaylydeco (ivacaftor) for the treatment of cystic fibrosis, tripling the number of rare gene mutations the drug is indicated to treat from 10 to 33.

Kalydeco is taken twice a day in either tablet or oral granule form with fat-containing food, and can be prescribed to patients 2 years and older with 1 of the mutations of cystic fibrosis that is responsive to treatment. If a patient’s genotype is unknown, a mutation test can be used to determine the mutation of disease.

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The extension of the drug to 33 genetic mutations of cystic fibrosis was based on results from an in vitro cell-based model system that reasonably predicts the clinical response of cystic fibrosis to treatment. Based on the efficacy of the drug in the laboratory model, the researchers were able to extrapolate its efficacy to humans by examining clinical benefits demonstrates in trials of other mutations.

Common adverse effects associated with Kalydeco include headache, upper respiratory tract infection (including the common cold, sore throat, nasal or sinus congestion, or runny nose), abdominal pain, diarrhea, rash, nausea, and dizziness. Kayldeco is also associated with increasing the risk for elevated transaminases and pediatric cataracts.

In addition, Kayldeco is not recommended to be taken with a strong CYP3A inducer as it has been shown to diminish the effectiveness of Kayldeco.

—Melissa Weiss

Reference:

FDA expands approved use of Kalydeco to treat additional mutations of cystic fibrosis [press release]. FDA. May 17, 2017. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm559212.htm?source=govdelivery&utm_medium=email&utm_source=govdelivery. Accessed May 18, 2017.