Vertex Gets EU Nod for New Cystic Fibrosis Drug Alyftrek

Core Insights - Vertex Pharmaceuticals (VRTX) received European Commission approval for Alyftrek (deutivacaftor/tezacaftor/vanzacaftor) to treat cystic fibrosis (CF) patients aged six and older with at least one non-class I mutation in the CFTR gene [1][2] - Alyftrek is a combination of three drugs and offers once-daily dosing, potentially providing better patient benefits compared to Trikafta [7][9] - The approval was anticipated following a positive opinion from the European Medical Agency's Committee for Medicinal Products for Human Use in April [2] Product and Market Position - Alyftrek is Vertex's fifth CFTR modulator therapy and was previously approved by the FDA in December 2024 and in the UK in March [2] - Regulatory filings for Alyftrek are under review in Canada, Switzerland, Australia, and New Zealand [3] - Vertex holds a dominant position in the CF market, with sales driven by the demand for Trikafta/Kaftrio, particularly in younger age groups [8] Financial Performance - Alyftrek generated sales of $53.9 million in Q1 2025, with expectations for stronger uptake in H2 2025 as patients switch from Trikafta [7][11] - Year-to-date, Vertex's shares have risen 12.3%, contrasting with a 3.4% decline in the industry [4] Clinical Efficacy - In head-to-head studies, Alyftrek demonstrated non-inferiority to Trikafta in improving ppFEV1 and showed superior reduction in sweat chloride levels, indicating better CFTR function [10] - Management believes Alyftrek could become a new standard-of-care treatment for CF and may treat patients who have discontinued Trikafta or other Vertex CF medicines [9] Future Outlook - Management anticipates that Alyftrek sales will increase as the majority of current Trikafta patients in the U.S. are expected to switch to Alyftrek [11] - Vertex is also evaluating Alyftrek for use in children with CF aged between two and five years [11]