Summary

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• Avalglucosidase alfa (Nexviazyme) is indicated for the treatment of patients 1 year of age and older with late-onset Pompe disease. It demonstrates a probable improvement in physical endurance or mobility, as evidenced by an increase in the 6-minute walk test distance compared to alglucosidase alfa.
• The study was derived from a systematic review/meta-analysis that evaluated six trials involving 358 randomised participants over periods ranging from 12 to 78 weeks.
• In terms of safety, avalglucosidase alfa shows little or no difference in infusion reactions and adverse events when compared to alglucosidase alfa. This indicates a favorable safety profile for avalglucosidase alfa.
• Compared to other therapies such as cipaglucosidase alfa with Miglustat, which may offer respiratory benefits, avalglucosidase alfa focuses on improving physical mobility while maintaining a comparable safety profile.
• While adjunct therapies were considered, avalglucosidase alfa provides more direct benefit without needing these additional treatments.
• Although the study does not delve into detailed subgroup analyses like age-specific or severity-specific responses due to its focus on late-onset Pompe disease across various ages; given LOPD's progressive nature, it suggests effectiveness and safety might vary across different subpopulations.