NEW YORK – The UK's Medicines and Healthcare Products Regulatory Agency (MHRA) has approved Vertex Pharmaceuticals' Alyftrek (vanzacaftor/tezacaftor/deutivacaftor) as a treatment for cystic fibrosis patients who are at least six years old and have certain genetic mutations, the company said on Friday.
Cystic fibrosis is a rare, progressive disease affecting the lungs and other organs and is caused by various genetic mutations in the CFTR gene, the most common of which is the F508del mutation. These mutations lead to missing or defective versions of the CFTR protein. Alyftrek, which Vertex previously has referred to as "vanza triple," is a CFTR modulator designed to increase the number of CFTR proteins and improve their function.
MHRA has specifically approved Boston-based Vertex's Alyftrek for cystic fibrosis patients at least six years old who have at least one F508del mutation or another responsive mutation in the CFTR gene.
The agency based its decision on two randomized Phase III studies that included 480 participants and showed that Alyftrek, a once-a-day oral medication, is as effective as the company's Kaftrio (ivacaftor/tezacaftor/elexacaftor), an already approved triple combination therapy, at improving lung function and reducing sweat chloride levels among cystic fibrosis patients. Kaftrio is sold in the US under the brand name Trikafta.
The most common side effects of Alyftrek were headache and diarrhea.
The US Food and Drug Administration approved Alyftrek in the same indication at the end of 2024, and it is under regulatory review in Europe, Canada, Switzerland, Australia, and New Zealand.