FDA OKs Kalydeco for Additional Mutations in Cystic Fibrosis

Share this content:
FDA OKs Kalydeco for Additional Mutations in Cystic Fibrosis
FDA OKs Kalydeco for Additional Mutations in Cystic Fibrosis

THURSDAY, May 18, 2017 (HealthDay News) -- The U.S. Food and Drug Administration says it has expanded approval for the cystic fibrosis medication Kalydeco (ivacaftor) to include 33 mutations of the disease, up from the previous 10 mutations.

In a media release, the agency said its decision was based on results of laboratory testing and previous clinical trials. Kalydeco is available as tablets or oral granules to be taken two times a day with fat-containing food.

Common side effects of the drug include headache, upper respiratory-tract infection, abdominal pain, diarrhea, rash, nausea, and dizziness. St. John's Wort and similar drugs and supplements could diminish Kalydeco's effectiveness and should be avoided, the FDA said.

"Many rare cystic fibrosis mutations have such small patient populations that clinical trial studies are not feasible," Janet Woodcock, M.D., director of the agency's Center for Drug Evaluation and Research, said in a statement. "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."

Kalydeco is produced by Boston-based Vertex Pharmaceuticals.

More Information

Share this content:

is free, fast, and customized just for you!

Already a member?

Sign In Now »

Drug Lookup

Browse drugs by: BrandGenericDisease

Trending Activities

All Professions



Sign up for myCME e-newsletters


More in Home

Surveillance Frequency Doesn't Cut Mortality in Colorectal Cancer

Surveillance Frequency Doesn't Cut Mortality in Colorectal Cancer

And, intensity of imaging surveillance not linked to time to detection of colorectal cancer recurrence

Procalcitonin Assay Doesn't Cut Antibiotic Use in Lower RTI

Procalcitonin Assay Doesn't Cut Antibiotic Use in Lower ...

Provision of assay doesn't result in less antibiotic use for suspected lower respiratory tract infection

Preventing Child Maltreatment Not Yet Feasible in Primary Care

Preventing Child Maltreatment Not Yet Feasible in Primary ...

USPSTF says evidence inadequate for primary care interventions to prevent child maltreatment

is free, fast, and customized just for you!

Already a member?

Sign In Now »