Vertex Expands FDA Approval For ALYFTREK And TRIKAFTA In Cystic Fibrosis, Broadens Access Covering 95% Of Patients

Vertex Pharmaceuticals Incorporated announced that the U.S. Food and Drug Administration has approved expanded use of ALYFTREK® (vanzacaftor/tezacaftor/deutivacaftor) for patients aged 6 and older with cystic fibrosis (CF) who have responsive CFTR gene variants. Additionally, the FDA broadened the indication for TRIKAFTA® to include patients as young as 2 years old.

The expanded approvals are supported by clinical and in vitro data across hundreds of CFTR variants, significantly increasing patient eligibility. Approximately 800 additional individuals in the U.S. can now access therapies that target the underlying cause of their disease for the first time.

“This groundbreaking approval represents more than 20 years of innovation in CF, including testing over 600 variants in our laboratory, demonstrating clinical effects in large clinical trials, and studying younger people with CF so they can be treated as early as possible,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. “With this label expansion, any variant that results in production of CFTR protein is now included in the ALYFTREK and TRIKAFTA labels, validating that these medicines can restore CFTR function and provide clinical benefit to patients regardless of where in the CFTR protein a variant is located. We thank the CF community and investigators for their trust and look forward to bringing ALYFTREK and TRIKAFTA to more patients than ever before.”

With these updates, around 95% of people living with cystic fibrosis in the U.S. are now eligible for treatment with CFTR modulators, marking a major step forward in expanding access to disease-modifying therapies.