Vertex Enters into Agreement with NHS England for New Cystic Fibrosis Treatment ALYFTREK®
Monday, July 14, 2025
Vertex Pharmaceuticals has reached a reimbursement agreement with NHS England for ALYFTREK® (deutivacaftor/tezacaftor/vanzacaftor), a once-daily triple combination therapy for eligible patients with cystic fibrosis.
Cystic fibrosis is a rare genetic condition that affects over 109,000 people worldwide, including around 94,000 in North America, Europe, and Australia. It is a progressive disease that impacts multiple organs, particularly the lungs, digestive system, liver, and reproductive tract.
The condition results from mutations in the CFTR gene, which lead to the production of a faulty or missing CFTR protein. This disrupts the flow of salt and water in and out of cells, causing thick, sticky mucus to build up—mainly in the lungs—leading to chronic infections and long-term lung damage.
CF is inherited when a child receives two defective copies of the CFTR gene, one from each parent. Although the disease is life-shortening, new therapies are helping to improve survival and quality of life. The most common mutation among patients is F508del.
Vertex’s CF therapies are now used by more than 75,000 people across over 60 countries. This includes approximately two-thirds of those diagnosed who are eligible for CFTR modulator treatments.
The new treatment combines three components: vanzacaftor, tezacaftor, and deutivacaftor. Vanzacaftor and tezacaftor help increase the amount of CFTR protein that reaches the cell surface, while deutivacaftor improves the function of the protein by keeping the channel open longer, allowing better salt and water transport. This targeted approach aims to address the underlying cause of CF at the cellular level.
The agreement follows a positive recommendation from NICE and marks another step in expanding access to innovative CF treatments across the UK.
ALYFTREK®, a once-daily triple combination therapy, is approved for use in patients aged six years and older who carry at least one F508del mutation or another responsive mutation in the CFTR gene. The treatment is considered a next-generation CFTR modulator and is Vertex’s fifth medicine targeting CF.
The new agreement with NHS England is expected to expand access for eligible patients across the country.
Vertex aims to secure similar agreements with health authorities across other EU member states to provide timely access to eligible patients.
Source: vrtx.com
