FX.co ★ Vertex's Cystic Fibrosis Drug Alyftrek Gets FDA Approval

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

Alyftrek is a cutting-edge, once-daily triple combination drug that modulates the cystic fibrosis transmembrane conductance regulator (CFTR) for patients aged six and older. It is specifically designed for individuals with at least one F508del mutation or other CFTR gene mutations that respond to Alyftrek.

"This approval marks our fifth CFTR modulator to receive FDA approval, representing a major milestone in our ongoing quest to bring innovative treatments to those with cystic fibrosis," stated Reshma Kewalramani, M.D., CEO and President of Vertex. "For over two decades, we have been committed to addressing the root cause of cystic fibrosis, expanding treatment options, and enhancing CFTR function for sufferers. Alyftrek, with its convenient once-daily dosing, efficacy across 31 additional mutations, and superior reductions in sweat chloride levels compared to TRIKAFTA, advances us further towards this goal."

The approval is based on data from the most comprehensive Phase 3 program ever conducted for cystic fibrosis, involving over 1,000 patients from more than 20 countries and 200 sites.

"In Phase 3 trials, across diverse genotypes, once-daily Alyftrek demonstrated non-inferiority in ppFEV1 response and a statistically significant improvement in sweat chloride levels compared to TRIKAFTA," noted Dr. Claire L. Keating, Co-Director of the Gunnar Esiason Adult Cystic Fibrosis and Lung Program at Columbia University and a key investigator in the Alyftrek clinical trials. "Alyftrek offers promising potential for enhancing patient care."

Alyftrek is also being reviewed by health authorities in the European Union, the United Kingdom, Canada, Switzerland, Australia, and New Zealand for potential approval.