Summary

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• This summary is based on the review of three randomized controlled trial(s). ^[1-3]
• Avalglucosidase alfa demonstrated superior effectiveness over alglucosidase alfa in improving health-related quality of life (HRQoL) in treatment-naive patients with late-onset Pompe disease (LOPD), with significant improvements observed in domains such as shortness of breath, fatigue, and overall activity levels (e.g., The Panic Disorder Severity Scale (PDSS), EQ-5D-5L, and Patient Global Impression of Change (PGIC) scores).
• In terms of respiratory function and physical endurance, avalglucosidase alfa led to a greater increase in forced vital capacity (FVC) percent predicted (2.89% vs. 0.46%) and 6-minute walk test distance (by 30.01 meters) compared to alglucosidase alfa over 49 weeks, with these improvements maintained or enhanced during the extension phase.
• Avalglucosidase alfa's overall effectiveness, including significant improvements in HRQoL and functional outcomes, positions it as a more favorable treatment option compared to alglucosidase alfa for LOPD patients.
• Treatment-emergent adverse events (TEAEs) were reported in 45% of patients in the avalglucosidase alfa group and 49% in the alglucosidase alfa group over 49 weeks, with infusion-associated reactions occurring in 26% and 33% of patients, respectively. Serious TEAEs were reported in 16% of avalglucosidase alfa patients compared to 25% in the alglucosidase alfa group.
• Antidrug antibody responses were observed in both groups, with high and persistent titers more common in the alglucosidase alfa group (33%) compared to the avalglucosidase alfa group (20%). No new safety concerns were identified with avalglucosidase alfa.
• The population studied primarily included treatment-naive patients with LOPD, ranging from 3 years of age and older. The safety and effectiveness outcomes, including improvements in respiratory function and endurance, are relevant to this broad patient population. No specific subgroup analyses or significant differences in effectiveness among different population types or subgroups were reported.