European Commission approves Alyftrek for cystic fibrosis
Action follows similar decisions in the US, UK
Alyftrek has been approved by the European Commission to treat cystic fibrosis (CF) patients 6 years and older who have at least one non-class 1 mutation in the CFTR gene.
This indication in the European Union (EU) represents the broadest label for Alyftrek (vanzacaftor/tezacaftor/deutivacaftor) in the world, according to the treatment’s developer, Vertex Phamaceuticals. Approval in the EU follows similar decisions in the U.S. and U.K.
“CF care has been transformed by the advent of highly effective CFTR modulators, and I am very pleased that we now have a new treatment option to even better address this [multisystemic] disease,” Marcus A. Mall, MD, a professor and chair of the Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine and Cystic Fibrosis Center at Charité Universitätsmedizin Berlin, said in a company press release.
Alyftrek is a next-generation, triple-combination CFTR modulator therapy designed to have similar or better efficacy, as well as more convenient dosing, than Vertex’s Trikafta (elexacaftor/tezacaftor/ivacaftor), which is sold as Kaftrio in the EU.
“Thousands of people with CF across the EU may now benefit from this new, once-daily medicine, which has demonstrated further improvement in CFTR protein function versus Kaftrio,” said Reshma Kewalramani, MD, president and CEO 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 CF.”
Alyftrek reduced chloride levels in sweat more than Trikafta
CF is caused by mutations in the CFTR gene, which contains instructions to make a protein of the same name. When the protein is missing or dysfunctional, the body manufactures mucus that is thick and sticky. This builds up in organs, producing most CF symptoms. Class 1 mutations result in little to no CFTR protein being produced
CFTR modulators bind to specific faulty versions of the CFTR protein to improve its functionality, allow the body to produce a more normal mucus, ease CF symptoms, and slow the progression of damage.
Alyftrek includes tezacaftor, which is also a component of Trikafta. It also includes vanzacaftor to help the CFTR protein fold correctly and reach the cell surface and deutivacaftor to make it more likely that the channel protein is open at the cell surface. Alyftrek is taken once daily, whereas Trikafta is given twice daily.
Three Phase 3 clinical trials have supported Alyftrek’s regulatory approvals: The completed SKYLINE 102 (NCT05033080) and SKYLINE 103 (NCT05076149) studies in CF patients 12 and older and an ongoing study (NCT05422222) in younger patients.
By bringing more people closer to normal level of CFTR function, this new medicine has the potential to further improve outcomes for patients.
Results from the trials in older patients showed Alyftrek reduced chloride levels in sweat more than Trikafta. This suggests a larger improvement in CFTR function. The two medications led to similar stabilization of lung function, with similar safety profiles.
Side effects of Alyftrek may include coughing, common cold, respiratory infections, headaches, throat pain, fatigue, rashes, and liver damage.
Vertex has reimbursement agreements in Ireland and Denmark, as well as agreements with countries including Germany to ensure healthcare system access to Alyftrek. The company expects individuals in these countries to have access to the medication soon. It plans to negotiate similar agreements in other EU member states.
“By bringing more people closer to normal level of CFTR function, this new medicine has the potential to further improve outcomes for patients,” Mall said.
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