Summary

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• This summary is based on the review of two systematic review(s)/meta-analysis(es). ^[1-2]
• In late-onset Pompe disease (LOPD), cipaglucosidase alfa with miglustat (cipa+mig) showed a significant increase in 6-minute walk distance (6MWD) and forced vital capacity (FVC) compared to avalglucosidase alfa (aval), with mean differences of 28.93 meters for 6MWD (95% confidence interval (CI): 8.26–50.11) and 2.88 percentage points for FVC (95% CI: 1.07–4.71). However, when limited to randomized control trials (RCTs), aval showed slightly better outcomes, with cipa+mig showing mean reductions of -10.02 meters for 6MWD (95% CI: -23.62 to 4.00) and -1.45 percentage points for FVC (95% CI: -3.01 to 0.07).
• In infantile-onset Pompe disease (IOPD), alglucosidase alfa significantly improved left ventricular (LV) mass with a mean change of 131.3 grams/m² (95% CI: 81.02, 181.59), delayed the time to start ventilation (hazard ratio (HR): 0.21; 95% CI: 0.12, 0.36), and enhanced survival (HR: 0.10; 95% CI: 0.05, 0.19), compared to the natural progression of the disease or placebo.
• Safety in LOPD: No specific safety data or adverse event information for cipaglucosidase alfa with miglustat (cipa+mig) or avalglucosidase alfa (aval) was provided.
• Safety in Infantile-Onset Pompe Disease (IOPD): Adverse events following enzyme replacement therapy (ERT) with alglucosidase alfa were generally mild.
• In LOPD, subgroup analysis suggested that cipaglucosidase alfa with miglustat (cipa+mig) demonstrated improved effectiveness in both 6MWD and FVC as the duration of prior ERT increased.