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.