Health Canada has approved avalglucosidase alfa (Nexviazyme™) for the long-term treatment of late-onset Pompe disease in patients over the age of 6 months.
Avalglucosidase alfa was approved by the FDA in August 2021. The FDA approval was based on positive results from the COMET clinical trial which compared avalglucosidase alfa to alglucosidase alfa in late-onset Pompe disease. These results demonstrated the drug’s safety, as the most frequently reported adverse reactions (>5%) in patients receiving avalglucosidase alfa were headache, diarrhea, itching, hives, and rash. Additionally, though 25% of the avalglucosidase alfa-treated patients experienced some kind of infusion related reactions, none of them were severe.
It is important to note that, while avalglucosidase alfa has been approved by Canada Health for the treatment of late-onset Pompe disease, this new treatment is not immediately accessible to individual patients in Canada. It is currently being reviewed by the Canadian Agency for Drugs and Technologies in Health (CADTH) and Institut national d’excellence en santé et en services sociaux (INESSS). Draft recommendations are expected in March, 2022. Individual provinces will also have to add avalglucosidase alfa to their health plans. Nevertheless, the approval of avalglucosidase alfa by Health Canada is a huge milestone for the Pompe disease community.