Vertex Pharmaceuticals’ triple combination cystic fibrosis (CF) treatment has been approved by the Medicines and Healthcare products Regulatory Agency (MHRA) for use in patients aged six years and older.
Patients eligible for Alyftrek (deutivacaftor/tezacaftor/vanzacaftor) will need to have at least one F508del mutation or another mutation in the CF transmembrane regulator (CFTR) gene that has been shown in trials to respond to the therapy.
More than 11,000 people in the UK are affected by CF, a rare inherited disease caused by a faulty CFTR gene, which helps regulate the flow of water and chloride in and out of the lungs and other organs.
In the lungs, this leads to the buildup of abnormally thick, sticky mucus, chronic infections and progressive damage that eventually leads to death for many patients.
Vertex’s Alyftrek is a once-daily oral CFTR modulator that works by correcting the malfunctioning protein made by the CFTR gene.
The MHRA’s approval of the therapy was based on positive results from two late-stage
clinical trials involving 480 CF patients aged 12 years and over.
Alyftrek was shown to be as effective at improving lung function as Vertex’s currently approved triple combination therapy Kaftrio (ivacaftor/tezacaftor/elexacaftor), and more effective at reducing sweat chloride levels.
Additional data from an open‑label phase 3 study also supported the regulator’s decision.
Carmen Bozic, executive vice president, global medicines development and medical affairs, and chief medical officer at Vertex, said: “For more than 20 years we have been focused on discovering medicines that treat the underlying cause of the disease with the goal of helping people live longer and better lives.
“The approval of Alyftrek… represents another significant milestone in that journey for people with CF in the UK.”
Vertex said it is working with the NHS and health technology assessment agency the National Institute for Health and Care Excellence (NICE) to ensure eligible patients can access the therapy “as soon as possible”.
David Ramsden, Cystic Fibrosis Trust chief executive, said: “[The] MHRA approval is another important step in making sure as many people with CF as possible can benefit from the best available treatments.
“We now hope that NICE will move quickly to complete its assessment of the medicine to enable it to be prescribed on the NHS.”
