Vertex Pharma’s Alyftrek is approved for patients 6 years and older with at least one responsive mutation, including 31 additional mutations not responsive to other CFTR modulator therapies.


RT’s Three Key Takeaways:

  1. FDA Approval of Alyftrek: The FDA has approved Alyftrek, a once-daily triple combination CFTR modulator, for people aged 6 and older with CF who have at least one responsive CFTR mutation.
  2. Successful Phase 3 Trials: Alyftrek met its primary and key secondary endpoints in extensive Phase 3 trials, showing non-inferiority to Trikafta in improving lung function and superior sweat chloride reduction.
  3. Broad Therapeutic Impact: Alyftrek was generally well tolerated and demonstrated efficacy across a wide range of genotypes, marking a significant advancement in cystic fibrosis treatment.


The US FDA has approved Alyftrek (vanzacaftor/tezacaftor/deutivacaftor), a once-daily next-in-class triple combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator. The therapy is approved for the treatment of cystic fibrosis (CF) in people 6 years and older who have at least one F508del mutation or another mutation in the CFTR gene that is responsive to Alyftrek.

“Alyftrek is our fifth CFTR modulator to secure FDA approval and represents another significant milestone in our journey to serially innovate and to improve the lives of people living with cystic fibrosis,” said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex.

This approval is based on the most comprehensive Phase 3 pivotal program ever conducted in CF, including more than 1,000 patients across more than 20 countries and more than 200 sites. These data were previously released at the conclusion of the studies and presented at the North American Cystic Fibrosis Conference in September of this year.

The Phase 3 studies in people with CF ages 12 years and older met their primary endpoint (non-inferiority on absolute change from baseline in ppFEV1 compared to Trikafta) and all key secondary endpoints (including absolute change from baseline in sweat chloride [SwCl] compared to Trikafta). In the Phase 3 study of children with CF ages 6-11 years, Alyftrek demonstrated safety, the primary endpoint. Secondary endpoints, such as absolute change from baseline in ppFEV1 and absolute change from baseline in SwCl, were presented, supporting the benefit of Alyftrek in this age group. Alyftrek was generally well tolerated across all studies.

“In Phase 3 clinical trials, across a broad range of genotypes, once-daily Alyftrek demonstrated non-inferiority to Trikafta in ppFEV1 response and statistically significant improvement in SwCl, a welcomed advancement for the treatment of CF,” said Claire L. Keating, MD, Co-Director of the Gunnar Esiason Adult Cystic Fibrosis and Lung Program at Columbia University and investigator in the Alyftrek clinical trial program. “Alyftrek has the potential to improve the care of patients with CF.”

See the Vertex website for Important Safety Information, including a Boxed Warning.