Vertex Pharmaceuticals announced the U.S. Food and Drug Administration approval of ALYFTREK, a once-daily triple combination treatment for cystic fibrosis (CF). This approval marks Vertex's fifth CFTR modulator, designed for individuals aged 6 and older with at least one F508del mutation or other responsive mutations in the CFTR gene. ALYFTREK, developed to improve CF treatment, demonstrated significant efficacy in Phase 3 trials, showing non-inferiority to TRIKAFTA and improved outcomes in key metrics, including sweat chloride levels. The drug’s once-daily dosing offers a more convenient regimen for patients, addressing a significant unmet need among the CF community.
The approval follows a comprehensive clinical program involving over 1,000 patients across 20 countries. Vertex’s Chief Executive Officer, Reshma Kewalramani, emphasized that ALYFTREK represents a significant step forward in the company’s mission to treat the underlying cause of CF. As the first once-daily CFTR modulator, ALYFTREK provides a potentially transformative option for patients, including those eligible for CFTR modulator therapy for the first time. Vertex is also pursuing global regulatory approvals, aiming to bring ALYFTREK to markets worldwide.
