CF treatment Alyftrek available to eligible patients in England
CFTR modulator aims to ease symptoms, slow disease progression
All eligible cystic fibrosis (CF) patients in England can now receive Vertex Pharmaceuticals’ CF treatment Alyftrek (vanzacaftor/tezacaftor/deutivacaftor), after the company reached a reimbursement agreement with the National Health Service (NHS).
The decision follows a positive recommendation from the National Institute for Health and Care Excellence and the treatment’s approval by the U.K. Medicines and Healthcare Products Regulatory Agency earlier this year for CF patients aged 6 and older who have at least one copy of a responsive mutation, including the most common F508del.
Eligible patients in Ireland, Denmark, and Germany will be the first in the European Union to access the treatment following Alyftrek’s recent EU approval. The company said it is working with other EU member states to ensure rapid access.
“We’re proud that ALYFTREK, our fifth CF medicine, is available today as another treatment option for all eligible CF patients in England,” Ludovic Fenaux, senior vice president of international commercial operations at Vertex, said in a company press release. “It represents a significant milestone in our journey to serially innovate and further improve the lives of people living with this disease.”
CF treatment combines medications
CF is caused by mutations in the CFTR gene, which provides instructions to produce a protein with the same name that controls the movement of water and salt molecules across cell membranes to produce watery mucus that provides protection and lubrication to the body’s tissues.
Mutations in the gene result in missing or dysfunctional CFTR protein, and in accumulation of the thick, sticky mucus that leads to most CF symptoms.
CFTR modulators bind to specific faulty versions of the CFTR protein to improve its function, allowing the body to produce more normal mucus, ease CF symptoms, and slow disease progression. Genetic testing conducted as part of CF diagnosis helps identify a patient’s specific mutations and determine which treatments may be effective.
Alyftrek combines three medications: tezacaftor and vanzacaftor, designed to help the CFTR protein fold correctly and reach the cell surface; and deutivacaftor, to increase the function of CFTR by making it more likely that the channel protein is open at the cell surface. Tezacaftor is also a component of Trikafta, a previously approved triple-combination CFTR modulator.
“In our pivotal studies, ALYFTREK demonstrated the potential for even better outcomes for patients than Kaftrio,” Trikafta’s brand name in the U.K., Fenaux said.
Alyftrek is intended to have a similar or higher efficacy than Kaftrio, and offers a more convenient, once-a-day dosing schedule. Kaftrio is given twice daily.
Results from Phase 3 clinical trials generally showed that Alyftrek was comparable to Trikafta at stabilizing lung function, with similar safety. It was also found to be more effective at reducing sweat chloride levels, indicating a higher effect at improving CFTR functionality.
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