Vertex Pharmaceuticals Secures UK MHRA Approval for ALYFTREK®, a Next-Generation CFTR Modulator for Cystic Fibrosis
Vertex Pharmaceuticals announced today that the United Kingdom (UK) Medicines and Healthcare products Regulatory Agency (MHRA) has granted approval for ALYFTREK® (deutivacaftor/tezacaftor/vanzacaftor), a next-in-class, once-daily triple combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator treatment. This newly approved therapy is indicated for individuals aged six years and older with cystic fibrosis (CF) who have at least one F508del mutation or another responsive mutation in the CFTR gene.
A Major Milestone in Cystic Fibrosis Treatment
Cystic fibrosis is a life-threatening genetic disorder caused by mutations in the CFTR gene that lead to the production of a defective CFTR protein. This protein is responsible for regulating the movement of chloride and water across cell membranes, and its dysfunction results in thick, sticky mucus buildup in the lungs, pancreas, and other organs. Over time, this buildup causes severe respiratory and digestive issues, increasing the risk of infections, lung damage, and other complications that significantly impact patients’ quality of life.
“For more than 20 years, we have been focused on discovering medicines that treat the underlying cause of CF, with the goal of helping people live longer and healthier lives,” stated Dr. Carmen Bozic, Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex Pharmaceuticals. “The approval of ALYFTREK, our fifth CFTR modulator regimen, represents another significant milestone in that journey for people with CF in the UK.”
Advancements in CFTR Modulation: What Sets ALYFTREK Apart?
ALYFTREK builds upon the success of previous CFTR modulator therapies, offering a once-daily dosing regimen designed to improve patient adherence and reduce treatment burden. Unlike earlier regimens requiring multiple daily doses, ALYFTREK provides a more convenient treatment approach while enhancing CFTR protein function.
According to clinical trial data, ALYFTREK demonstrated superior efficacy in improving lung function and reducing sweat chloride levels compared to existing CFTR modulator combinations. The pivotal Phase 3 trials revealed that children and adults treated with deutivacaftor/tezacaftor/vanzacaftor were more likely to achieve sweat chloride levels comparable to those of CF carriers.
“Children and adults taking the new triple combination therapy were more likely to have carrier levels of sweat chloride compared to those on the ivacaftor/tezacaftor/elexacaftor regimen, which we hope will translate to a reduced risk of developing CF-related complications in the long term,” stated Professor Alex Horsley, Professor of Respiratory Medicine at the University of Manchester, UK. “This once-daily, more flexible regimen represents a major step forward in the management of CF, particularly in improving treatment adherence and overall patient outcomes.”
The Impact of ALYFTREK on Patients and Healthcare Systems
For individuals living with CF, access to effective and less burdensome treatments is essential for improving quality of life and long-term health outcomes. The approval of ALYFTREK provides a new therapeutic option that aims to enhance lung function, reduce exacerbations, and slow disease progression.
Vertex Pharmaceuticals is actively working with the National Institute for Health and Care Excellence (NICE) and the UK’s National Health Service (NHS) to facilitate swift patient access to ALYFTREK. Discussions with regulatory and healthcare bodies are essential to ensure the therapy becomes widely available without delay.
“Ensuring patients can access innovative treatments like ALYFTREK as quickly as possible is a top priority,” a Vertex spokesperson emphasized. “We are committed to working closely with the NHS to integrate this therapy into treatment protocols and expand availability to all eligible patients.”
Regulatory Status and Global Availability
The UK MHRA’s approval of ALYFTREK follows similar regulatory advancements worldwide. In December 2024, the U.S. Food and Drug Administration (FDA) granted approval for ALYFTREK for individuals aged six years and older with at least one responsive CFTR mutation. Marketing Authorization Applications (MAAs) are also under review with the European Medicines Agency (EMA) and regulatory authorities in Canada, Switzerland, Australia, and New Zealand. These ongoing evaluations underscore the global commitment to expanding treatment options for people living with CF.
The Future of CF Treatment and Ongoing Research
The development of ALYFTREK is part of Vertex Pharmaceuticals’ broader commitment to advancing CF care. While CFTR modulators have transformed treatment paradigms for many patients, ongoing research continues to explore new approaches, including genetic and cell-based therapies aimed at providing a potential cure for CF.
Vertex is investing in innovative technologies such as gene editing and mRNA therapies to further address the underlying genetic cause of CF. By leveraging cutting-edge research, the company aims to develop novel treatments that could benefit patients who do not respond to existing CFTR modulators.
“The approval of ALYFTREK represents another critical step forward, but we remain committed to pushing the boundaries of CF research,” Dr. Bozic added. “Our long-term goal is to develop curative therapies that will one day eliminate the burden of this disease for all patients.”
