BOSTON — Vertex Pharmaceuticals Incorporated said the U.S. Food and Drug Administration has approved expanded indications for its cystic fibrosis therapies ALYFTREK and TRIKAFTA, significantly increasing the number of patients eligible for treatment.
The updated labels allow ALYFTREK to be used in patients ages 6 and older with cystic fibrosis who have gene variants that either respond to treatment based on clinical or laboratory data or result in production of the CFTR protein. The FDA also expanded the indication for TRIKAFTA to include patients as young as 2 years old.
Vertex said the decision means roughly 800 additional people in the U.S. with cystic fibrosis are now eligible for a CFTR modulator therapy for the first time. Overall, about 95% of cystic fibrosis patients in the country can now be treated with one of these medicines.
The approvals were supported by data evaluating hundreds of genetic variants, including 564 variants responsive to ALYFTREK and 521 variants responsive to TRIKAFTA.
“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.”
Cystic fibrosis is a genetic disease caused by mutations in the CFTR gene, leading to thick mucus buildup in the lungs and other organs. CFTR modulators such as ALYFTREK and TRIKAFTA target the underlying cause of the disease by improving the function of the defective protein.
The expanded approvals mark a significant step in extending treatment to nearly all cystic fibrosis patients in the U.S., including those with rare or previously untreated genetic variants.
