Vertex's Cystic Fibrosis Drug Alyftrek Gets FDA Approval

(RTTNews) - Vertex Pharmaceuticals Inc. (VRTX) Friday announced that the U.S. Food and Drug Administration (FDA) has approved Alyftrek for treatment of cystic fibrosis.

Alyftrek is a once-daily next-in-class triple combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator 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. "Our north star for more than 20 years has been to address the underlying cause of cystic fibrosis, treat more people with this disease, and bring more people to normal levels of CFTR function — ALYFTREK, with once-daily dosing, efficacy in 31 additional mutations, and lower sweat chloride levels than TRIKAFTA, is another step in achieving this goal."

This approval is based on the most comprehensive Phase 3 pivotal program ever conducted in cystic fibrosis, including more than 1,000 patients across more than 20 countries and more than 200 sites.

"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, M.D., 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."

Alyftrek was also submitted to global health authorities and is under regulatory review in the European Union, the United Kingdom, Canada, Switzerland, Australia and New Zealand.