Vertex Pharmaceuticals announced that Health Canada has granted Marketing Authorization for PrALYFTREKTM (vanzacaftor/tezacaftor/deutivacaftor), a new triple combination therapy for patients living with cystic fibrosis (CF) ages 6 years and older who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
ALYFTREK brings a potentially transformative therapy to up to 60 people living with CF in Canada who were not previously eligible for a CFTR modulator. As the first once-daily CFTR modulator, ALYFTREK may also provide a new treatment option and flexibility for approximately 3,800 people given the need to take CFTR modulators with fat-containing food.
"ALYFTREK represents the next generation of CFTR modulator treatment and is a testament to our long-standing 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. "With once-daily dosing, eligibility in 113 additional mutations, and the potential to lower sweat chloride levels even further, ALYFTREK brings us one step closer to achieving this goal."
This approval is based on a comprehensive Phase 3 pivotal program, including more than 1,000 patients across more than 20 countries and more than 200 sites. The Phase 3 studies in people with CF ages 12 years and older met their primary endpoint (non-inferiority on absolute change from baseline in ppFEV1 compared to TRIKAFTA) and all key secondary endpoints (including absolute change from baseline in sweat chloride [SwCl] compared to TRIKAFTA). In the Phase 3 study of children with CF ages 6-11 years, ALYFTREK demonstrated safety, the primary endpoint. Secondary endpoints, such as absolute change from baseline in ppFEV1 and absolute change from baseline in SwCl, demonstrate the benefit of ALYFTREK in this age group. ALYFTREK was generally well tolerated across all studies.
"For Canadian patients and families, the approval of ALYFTREK represents significant progress towards improved care," said Dr. Bradley Quon, Medical and Research Director of the Adult Cystic Fibrosis Program at St. Paul's Hospital, and Associate Professor of Medicine in the Faculty of Medicine at the University of British Columbia. "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."
ALYFTREK is currently approved in the U.S., UK and European Union and is under regulatory review in Switzerland, Australia and New Zealand.
Subscribe to our e-Newsletters
Stay up to date with the latest news, articles, and events. Plus, get special offers
from American Pharmaceutical Review – all delivered right to your inbox!
Sign up now!