FDA Approves ALYFTREK, a Next-Generation CFTR Modulator for Cystic Fibrosis Treatment
FDA Approval:
The U.S. FDA has approved ALYFTREK, a once-daily triple combination CFTR modulator developed by Vertex Pharmaceuticals, for the treatment of cystic fibrosis in patients 6 years and older with at least one responsive mutation12.
Clinical Trials:
The approval is based on comprehensive Phase 3 trials involving over 1,000 patients across 200+ sites in 20+ countries, demonstrating non-inferiority to TRIKAFTA on ppFEV1 and improved sweat chloride levels25.
Expanded Coverage:
ALYFTREK extends treatment to 31 additional mutations not previously responsive to other CFTR modulators, potentially benefiting approximately 150 additional CF patients in the U.S.12.
Safety Information:
The drug comes with a boxed warning for potential drug-induced liver injury, including liver failure5.
Market Impact:
This approval strengthens Vertex's dominant position in CF treatment and expands their addressable market, with ALYFTREK forecasted to bring in $5.3 billion in sales by 20305.
Sources:
1. https://investors.vrtx.com/news-releases/news-release-details/vertex-announces-us-fda-approval-alyftrektm-once-daily-next
2. https://www.stocktitan.net/news/VRTX/vertex-announces-us-fda-approval-of-alyftrektm-a-once-daily-next-in-69ja9jhbikdw.html
5. https://www.pharmaceutical-technology.com/news/vertex-secures-two-fda-wins-for-cystic-fibrosis-both-with-boxed-warnings/