FDA Approves Vertex's Cystic Fibrosis Therapy

The FDA approval of Vertex’s ALYFTREK, a once-daily cystic fibrosis therapy, reinforces the company’s leadership in managing the disease. Alyftrek offers a new option for patients aged six and older, ensuring continued treatment innovation. 

“ALYFTREK is our fifth CFTR modulator to secure FDA approval and represents another significant milestone in our journey to serially innovate and to improve the lives of people living with cystic fibrosis,” said Reshma Kewalramani, CEO and President, Vertex, via the company’s press release. “Our north star for more than 20 years has been to address the underlying cause of cystic fibrosis, treat more people with this disease, and bring more people to normal levels of CFTR function. ALYFTREK, with once-daily dosing, efficacy in 31 additional mutations, and lower sweat chloride levels than TRIKAFTA, is another step in achieving this goal.”

ALYFTREK combines vanzacaftor, tezacaftor, and deutivacaftor and is set to launch in January 2025 with an annual wholesale acquisition cost of US$370,269. The approval marks a significant milestone in Vertex’s CF portfolio, which includes the top-selling drug TRIKAFTA, writes Reuters. 

In late-stage clinical trials, ALYFTREK demonstrated non-inferiority to Trikafta, which is taken twice daily. This new therapy aims to sustain Vertex's market leadership as TRIKAFTAapproaches patent expiration.

Cystic fibrosis is a progressive genetic condition which results in the buildup of thick, sticky mucus in the lungs and other organs, leading to breathing difficulties and potential lung damage. Vertex’s treatments have transformed this disease from life-threatening to a manageable condition for many patients, significantly extending life expectancy.