Vertex Receives Approval for ALYFTREK® to Treat Cystic Fibrosis
Wednesday, July 02, 2025
Vertex Pharmaceuticals has announced that the European Commission has approved ALYFTREK® (deutivacaftor/tezacaftor/vanzacaftor), a new once-daily CFTR modulator, for the treatment of cystic fibrosis (CF) in patients aged six years and above with at least one non-class I mutation in the CFTR gene.
ALYFTREK® combines vanzacaftor and tezacaftor to support CFTR protein processing and trafficking, while deutivacaftor acts to increase channel activity, helping restore salt and water flow across cell membranes.
Sweat chloride (SwCl) concentration is used to diagnose CF and assess CFTR function. Lower SwCl levels are linked to improved clinical outcomes such as better lung function and reduced complications. ALYFTREK® has shown the ability to lower SwCl to levels closer to those of individuals without CF, representing a significant step forward in treatment efforts.
The approval follows two pivotal clinical trials where ALYFTREK® demonstrated non-inferiority to KAFTRIO® in combination with ivacaftor on ppFEV1, while showing superior reduction in sweat chloride levels. These findings suggest improved CFTR protein function compared to existing treatments.
The introduction of ALYFTREK® is expected to expand treatment options for individuals with CF, a genetic condition affecting over 109,000 people worldwide. CF impacts multiple organs, including the lungs, liver, pancreas, and reproductive system, and is caused by mutations in the CFTR gene that result in reduced or defective CFTR protein. This leads to thick mucus build-up, especially in the lungs, often resulting in chronic infections and progressive lung damage.
Vertex is actively working with other EU member states to facilitate access across the region.
Currently, Vertex CF medicines are being used by more than 75,000 people across over 60 countries, representing around two-thirds of the global CF population eligible for CFTR modulator therapy.
Source: vrtx.com
