England’s National Institute for Health and Care Excellence issued a positive final draft recommendation for Vertex Pharma’s Alyftrek CFTR modulator, which will now be available via the National Health Service (NHS).
Vertex Pharmaceuticals Inc has reached a broad reimbursement agreement with NHS England for Vertex’s cystic fibrosis (CF) medicine Alyftrek (deutivacaftor/tezacaftor/vanzacaftor). This agreement comes as the National Institute for Health and Care Excellence (NICE) has issued a positive final draft recommendation for this medicine, according to the company.
This next-in-class triple combination treatment is licensed for people living with CF aged 6 years and older who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
“We’re proud that Alyftrek, our fifth CF medicine, is available today as another treatment option for all eligible CF patients in England. It represents a significant milestone in our journey to serially innovate and further improve the lives of people living with this disease,” said Ludovic Fenaux, Senior Vice President, Vertex International. “In our pivotal studies, Alyftrek demonstrated the potential for even better outcomes for patients than Kaftrio (ivacaftor/tezacaftor/elexacaftor). We’re pleased to have reached this agreement with NHS England that recognizes the value that this new medicine brings to CF patients, their families and society.”
Following the European regulatory approval, eligible patients in Ireland, Denmark and Germany will be the first ones to access deutivacaftor/tezacaftor/vanzacaftor in the European Union. Vertex will continue to work with reimbursement bodies across the European Union member states to ensure access for all eligible patients as quickly as possible.
Cystic fibrosis is a rare, life-shortening genetic disease affecting more than 109,000 people, including 94,000 people in North America, Europe and Australia. CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands, and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF, and these mutations can be identified by a genetic test. While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation. CFTR mutations lead to CF by causing CFTR protein to be defective or by leading to a shortage or absence of CFTR protein at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. 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.
In people with CF, mutations in the CFTR gene lead to decreased quantity and/or function of the CFTR protein channel at the cell surface. Vanzacaftor and tezacaftor are designed to increase the amount of CFTR protein at the cell surface by facilitating the processing and trafficking of the CFTR protein. Deutivacaftor is a potentiator designed to increase the channel open probability of the CFTR protein delivered to the cell surface to improve the flow of salt and water across the cell membrane.
