Regulatory Decision Summary for Nexviazyme

The sponsor for Nexviazyme (avalglucosidase alfa for injection) was based on results of a multicentre, multinational, randomized, phase 3 pivotal study, in subjects with late onset Pompe disease (LOPD) with support from a phase a Phase 2, multi-stage, open-label, multicentre, multinational, repeated ascending-dose cohort study of avalglucosidase alfa in pediatric subjects with IOPD previously treated with alglucosidase alfa (AGA). In the pivotal study, treatment with Nexviazyme showed non-inferiority in terms of % forced vital capacity (FVC) compared to the current standard of therapy. The secondary efficacy endpoints were supportive of a clinical benefit. While a favourable trend was observed, the results were considered to be exploratory.

The most common adverse events for all subjects included nasopharyngitis, headache, diarrhea, back pain, fall and nausea.  Important adverse events of special interest were hypersensitivity and anaphylactic reactions and infusion associated reactions.  Adverse reactions of Nexviazyme were generally manageable and consistent with the established safety profile of AGA. The safety of Nexviazyme is adequately described in the final product monograph. A Risk Management Plan (RMP) was assessed and considered acceptable by Health Canada.

Overall, the benefit-risk profile of Nexviazyme for the treatment of patients with Pompe disease was favourable for the subpopulation of subjects with LOPD, however, the results were insufficient to recommend an indication in IOPD.

For more information on Health Canadas decision, please view the Summary Basis of Decision.