Vertex Gains US FDA Approval for ALYFTREK™, a Daily CFTR Modulator for Cystic Fibrosis Treatment

Vertex Pharmaceuticals has achieved a notable advancement in the treatment of cystic fibrosis (CF) with the recent approval of ALYFTREK by the U.S. Food and Drug Administration (FDA). ALYFTREK, a novel triple combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator, is approved for patients aged six and older with at least one F508del mutation or another mutation in the CFTR gene responsive to this treatment. This new therapy is significant as it addresses 31 additional mutations that were previously unresponsive to other CFTR modulator therapies.

The approval of ALYFTREK is a major milestone for Vertex Pharmaceuticals, marking their fifth CFTR modulator to receive FDA approval. Reshma Kewalramani, the Chief Executive Officer of Vertex, emphasized the company's commitment to improving the lives of people with CF by addressing the underlying causes of the disease and enhancing CFTR function. ALYFTREK offers a once-daily dosing regimen and has demonstrated efficacy in reducing sweat chloride levels more effectively than the existing drug, TRIKAFTA.

The FDA's approval was based on extensive Phase 3 clinical trials, which included more than 1,000 CF patients from over 20 countries. These studies demonstrated that ALYFTREK was non-inferior in terms of ppFEV1, a measure of lung function, compared to TRIKAFTA. Additionally, ALYFTREK showed a significant reduction in sweat chloride levels in patients, indicating improved CFTR function. The trials also confirmed the safety and tolerability of ALYFTREK in both children and adults with CF.

Dr. Claire L. Keating, a key investigator in the ALYFTREK clinical trials, highlighted the therapy's potential to enhance CF care. The once-daily dosage addresses a significant unmet need for CF patients, allowing for more convenient administration alongside fat-containing foods necessary for CFTR modulator absorption.

Globally, Vertex has submitted ALYFTREK for regulatory review in multiple regions, including the European Union and Canada, among others. The company aims to make this promising therapy available to a wider population of CF patients.

Cystic fibrosis is a rare, life-shortening genetic disorder affecting multiple organs, particularly the lungs. It is caused by defects in the CFTR protein, which leads to the accumulation of thick mucus, resulting in chronic lung infections and progressive respiratory damage. The majority of CF patients have at least one F508del mutation, making them potential candidates for ALYFTREK treatment.

Vertex Pharmaceuticals remains at the forefront of developing transformative medicines for serious genetic diseases. With the introduction of ALYFTREK, Vertex continues its mission to innovate and provide effective treatments for conditions like cystic fibrosis. The company's dedication to scientific research and breakthrough therapies has positioned them as a leader in biotechnology, with a strong global presence and a commitment to improving patient outcomes worldwide.