FDA
FDA expands CF indications for Alyftrek and Trikafta
On April 1, 2026, FDA approved label expansions for CFTR modulators Alyftrek (vanzacaftor/tezacaftor/deutivacaftor) and Trikafta (elexacaftor/tezacaftor/ivacaftor), broadening access to treatment for people with cystic fibrosis (CF). The updated indications now include any CFTR gene variant that results in production of CFTR protein, regardless of mutation location, based on clinical and/or in vitro responsiveness data.
Under the revised labels, Alyftrek is indicated for patients aged ≥6 years, and Trikafta for those aged ≥2 years. The decision was supported by data from 564 CFTR variants for Alyftrek and 521 variants for Trikafta, making an estimated ~800 additional patients in the U.S. newly eligible for a CFTR modulator and increasing overall eligibility to ~95% of the U.S. CF population.
Commenting on the approval in company news release, Vertex Chief Medical Officer Carmen Bozic, MD, noted that the updated labels reflect evidence that these therapies can provide benefit “regardless of where in the CFTR protein a variant is located.”