FDA approves Alyftrek for cystic fibrosis treatment

The FDA has approved Alyftrek for patients aged 6 years and older with cystic fibrosis with certain mutations, according to a press release from Vertex Pharmaceuticals.

This approval applies to patients in this age range who possess at least one F508del mutation in the CFTR gene or a mutation in the CFTR gene that responds to Alyftrek (vanzacaftor/tezacaftor/deutivacaftor, Vertex Pharmaceuticals), according to the release.

The triple combination modulator received FDA approval based, in part, on data from three phase 3 trials — two involving patients aged 12 years and older and one involving patients aged 6 to 11 years.

According to an earlier press release from Vertex Pharmaceuticals, these studies assessed use of the therapy taken once a day over 52 weeks in the older population or 24 weeks in the younger population vs. Trikafta (elexacaftor/tezacaftor/ivacaftor, Vertex Pharmaceuticals).

The trials found that the patients receiving Alyftrek generally tolerated it well. Additionally, the release said that researchers observed improved sweat chloride and “non-inferiority” in percent predicted FEV₁ with this combination vs. Trikafta.

“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,” Reshma Kewalramani, MD, CEO and president of Vertex, said in the 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.”

Reference:

• Vertex announces positive results from pivotal trials of vanzacaftor/tezacaftor/deutivacaftor, next-in-class triple combination treatment for cystic fibrosis. https://investors.vrtx.com/news-releases/news-release-details/vertex-announces-positive-results-pivotal-trials. Published Feb. 5, 2024. Accessed Dec. 23, 2024.