Alyftrek approved in Canada for CF patients with responsive mutations
People 6 years and older are eligible for the CFTR modulator therapy
Health Canada has approved the next-generation CFTR modulator therapy Alyftrek for people 6 years and older with cystic fibrosis (CF) who have at least one mutation that’s responsive to the therapy. That includes the most common CF-causing mutation, F508del.
The approval in Canada follows similar approvals in the U.S., the U.K., and the European Union. Alyftrek is currently under regulatory review in Switzerland, Australia, and New Zealand, according to its maker, Vertex Pharmaceuticals.
“For Canadian patients and families, the approval of [Alyftrek] represents significant progress [toward] improved care,” Bradley Quon, MD, an associate professor at the University of British Columbia, said in a press release from Vertex.
Alyftrek developed as Trikafta successor
CF is caused by mutations in the gene that provides instructions to make CFTR, a protein that helps regulate the production of mucus. In CF, defective or absent CFTR leads to the production of abnormally thick and sticky mucus, which builds up in organs to drive most disease symptoms.
CFTR modulators are medications that can boost the functionality of the faulty CFTR protein in people with specific mutations. Alyftrek is a combination of three modulator molecules, vanzacaftor, tezacaftor, and deutivacaftor. It was developed as a next-generation successor to Trikafta (elexacaftor/tezacaftor/ivacaftor), another triple-combination modulator therapy made by Vertex that is widely approved to treat CF in people with F508del and other responsive mutations.
“Alyftrek represents the next generation of CFTR modulator treatment and is a testament to our [longstanding] goal to address the underlying cause of cystic fibrosis, treat more people living with CF, and bring more people to normal levels of CFTR function,” said Michael Siauw, general manager at Vertex Pharmaceuticals (Canada) Incorporated.
Alyftrek was compared against Trikafta in two Phase 3 clinical trials: the SKYLINE 103 (NCT05076149) and the SKYLINE 102 (NCT05033080) studies in patients ages 12 and older. Another study, (NCT05422222), in children ages 1 to 11, will have all participants receive the investigative treatment. It is still enrolling at sites in North America, Europe, Australia and New Zealand.
Lower levels of sweat chloride combined with the convenience of a once-a-day treatment provides a new option that has the potential to both improve CFTR function and reduce treatment burden.
People with CF characteristically have high levels of chloride, a component of table salt, in their sweat due to CFTR protein dysfunction. Clinical trial data indicated Alyftrek is better than Trikafta at reducing sweat chloride levels, implying that the next-generation therapy is more potent for boosting CFTR protein function. Both therapies showed comparable ability to preserve lung function.
Alyftrek is taken once daily alongside a fat-containing food like peanut butter, meat, or cheese. The therapy’s common side effects include cough, respiratory infections, headache, fatigue, rash, and elevated liver enzyme levels.
“Lower levels of sweat chloride combined with the convenience of a once-a-day treatment provides a new option that has the potential to both improve CFTR function and reduce treatment burden,” Quon said.
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