Trikafta Receives Expanded Cystic Fibrosis Approval

Trikafta Receives Expanded Cystic Fibrosis Approval

June 10, 2021 – The U.S. FDA has approved an expanded indication for Trikafta® (elexacaftor/tezacaftor/ ivacaftor), manufactured by Vertex Pharmaceuticals. The drug is now indicated to treat cystic fibrosis in patients who are at least six years old and have at least one F508del mutation in the CFTR gene or a mutation in the CFTR gene that is responsive based on in vitro data. Previously, Trikafta was approved only for patients ages 12 years and up.

Cystic fibrosis is a progressive disease that affects the body’s ability to attract water to cell surfaces. This leads to the creation of abnormally thick, sticky mucus in and around various organs, which compromises proper organ function and increases the risk of infection. Cystic fibrosis can affect breathing, growth, nutrient absorption, and other regular functions of the body. An estimated 30,000 individuals in the United States have cystic fibrosis, and about 1,000 new cases are identified annually. Most new patients are diagnosed before they reach two years of age.

In clinical trials, children 6-11 years old who received Trikafta to treat cystic fibrosis had improvements in lung function and quality of life. Trikafta’s safety profile in this age group was comparable to that observed in older patient populations.

Recommended dosing for children six to less than 12 years old is based on weight and requires both a morning and evening dose of medication. Trikafta should be taken by mouth with fat-containing food. It should not be used in patients diagnosed with severe hepatic impairment, and it is not recommended for use in patients who have moderate hepatic impairment unless the benefit outweighs the risk. Trikafta first received FDA approval in 2019.