NEW YORK – The US Food and Drug Administration has approved Vertex Pharmaceuticals' Alyftrek (vanzacaftor/tezacaftor/deutivacaftor) as a cystic fibrosis treatment for patients at least six years of age and with certain genetic mutations.
Cystic fibrosis is a rare disease caused by various mutations in the CFTR gene that lead to defective or missing CFTR proteins. Alyftrek, an oral medication that's taken daily, is a CFTR modulator designed to increase the quantity and improve the function of the CFTR protein in patients with cystic fibrosis. Vertex previously referred to it as "vanza triple."
The FDA on Friday approved Alyftrek based on data from a pivotal Phase III trial, which demonstrated non-inferiority on absolute change from baseline in percent predicted forced expiratory volume, a measure designed to illustrate lung function, compared to Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor), another CFTR modulator developed by Vertex.
In a separate Phase III trial in pediatric patients, investigators found that Alyftrek was safe, which was the study's primary endpoint.
"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," Vertex CEO and President Reshma Kewalramani said in a statement. He noted that the label includes 31 genetic mutations that aren't responsive to other CFTR modulators.
The Alyftrek label includes a boxed warning for the risk of liver injury and liver failure.
Alyftrek is under regulatory review in the EU, UK, Canada, Switzerland, Australia, and New Zealand.
Separately, the FDA expanded the indication for Vertex's Trikafta on Friday to include cystic fibrosis patients with additional genetic variants.
The FDA initially approved Trikafta in 2019 for patients at least 12 years of age with at least one F508del mutation in the CFTR gene, which is the most common type of cystic fibrosis mutation, and since then has expanded the label multiple times to include younger patients and additional variants.
The FDA has now approved the expanded use of Trikafta as a treatment for cystic fibrosis patients who have one of 94 other genetic mutations that are responsive to the drug based on clinical or in vitro data. Boston-based Vertex estimated that the label expansion will make Trikafta available to about 300 additional cystic fibrosis patients in the US.
The FDA also added a boxed warning regarding liver injury and liver failure, which previously had been included in the warnings and precautions section of the drug's label.
"With this approval, even more patients may be able to benefit from a medicine that treats the underlying cause of their disease, and we look forward to continuing the work to extend the approvals and availability of our medicines to patients around the world," Carmen Bozic, Vertex's chief medical officer and executive VP of global medicines development and medical affairs, said in a statement.