Summary

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• Nexviazyme (avalglucosidase alfa) is indicated for the treatment of patients 1 year of age and older with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency).
• This summary is based on the review of one systematic review(s)/meta-analysis(es). ^[1]
• Alglucosidase Alfa vs. Placebo: Alglucosidase alfa probably improves the 6MWT distance (MD 30.95 meters, 95% CI 7.98 to 53.92) and respiratory function (MD 3.55% predicted FVC, 95% CI 1.46 to 5.64) compared to placebo, with moderate-certainty evidence. There may be little or no difference in quality of life physical component score (MD -1.36 points, 95% CI -5.59 to 2.87; low-certainty evidence).
• Cipaglucosidase Alfa plus Miglustat vs. Alglucosidase Alfa plus Placebo: Cipaglucosidase alfa plus miglustat probably improves % predicted FVC compared to alglucosidase alfa plus placebo (MD 3.10%, 95% CI 1.04 to 5.16; moderate-certainty evidence). It may make little or no difference in the 6MWT distance (MD 13.60 meters, 95% CI -2.26 to 29.46) or quality of life scores for physical function and fatigue, with low-certainty evidence.
• Avalglucosidase Alfa vs. Alglucosidase Alfa: Avalglucosidase alfa probably improves 6MWT distance compared to alglucosidase alfa (MD 30.02 meters, 95% CI 1.84 to 58.20) with moderate-certainty evidence, but likely makes little or no difference to % predicted FVC (MD 2.43%, 95% CI -0.08 to 4.94; moderate-certainty evidence).
• Alglucosidase Alfa vs. Placebo: There may be little or no difference in the risk of infusion reactions (RR 1.21, 95% CI 0.57 to 2.61) or adverse events (RR 0.94, 95% CI 0.64 to 1.39; low-certainty evidence).
• Avalglucosidase Alfa vs. Alglucosidase Alfa: There may be little or no difference in the risk of infusion reactions (RR 0.78, 95% CI 0.42 to 1.45) or adverse events (RR 0.92, 95% CI 0.61 to 1.40; low-certainty evidence).
• There is no population types or subgroups information available in the reviewed studies.