HQ Team
July 3, 2025: The European Commission has approved Vertex Pharmaceuticals‘ drug to treat cystic fibrosis in adults and children above six years of age who have certain genetic mutations related to the disease.
The European Medicines Agency, the region’s regulator, cleared the once-daily triple combination therapy, branded Alyftrek, for patients with the rare and progressive genetic disease, according to a company statement.
The medicine helps improve the function of a faulty protein, called CFTR or cystic fibrosis transmembrane conductance regulator, that causes cystic fibrosis.
Children must inherit two defective CFTR genes — one from each parent — to have cystic fibrosis, and these mutations can be identified by a genetic test.
Lungs, other organs
Cystic fibrosis is a genetic disease that affects the lungs and other organs. Alyftrek works by fixing the CFTR protein so it can function more normally, which can improve lung function and reduce symptoms.
In clinical trials, it performed as well as or better than an existing treatment called Kaftrio, especially in reducing sweat chloride levels—a key sign of how well the CFTR protein is working.
Countries like Ireland and Denmark have already agreed to cover the cost of Alyftrek, and Vertex is working to make it available to eligible patients throughout the European Union as soon as possible.
It is now approved in the EU, the US, and the UK, with more countries reviewing it for use. Alyftrek got the US approval last year.
The approval follows a positive recommendation from the European Medicines Agency (EMA), the region’s drug regulator. The European Commission, which normally follows the EMA’s guidance, gives final approval for new drugs.
CFTR protein function
“Thousands of people with cystic fibrosis across the EU may now benefit from this new, once-daily medicine, which has demonstrated further improvement in CFTR protein function versus,” said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex.
“With this approval, we are one step closer to our ultimate goal of restoring normal levels of CFTR function in people living with cystic fibrosis.”
Cystic fibrosis, a rare, life-shortening genetic disease, affects more than 109,000 people, including 94,000 people in North America, Europe and Australia. It is a progressive, multi-organ disease that affects the lungs, liver, pancreas, gastrointestinal tract, sinuses, sweat glands and reproductive tract.
The defective function or absence of the CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs.
Sticky mucus
In the lungs, this leads to the buildup of abnormally thick, sticky mucus, chronic lung infections and progressive lung damage that eventually leads to death for many patients.
The median age of death is in the 30s, but with treatment, projected survival is improving.
“Deutivacaftor/tezacaftor/vanzacaftor has shown it can deliver greater reductions in sweat chloride compared to standard of care,” said Professor Marcus A. Mall, M.D., Professor and Chair of the Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine and Cystic Fibrosis Center at Charité Universitätsmedizin Berlin.
“By bringing more people closer to the normal level of CFTR function, this new medicine has the potential to further improve outcomes for patients.”
