Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naïve and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascending dose study.

•Avalglucosidase alfa (neoGAA) had a well-tolerated safety profile in LOPD patients.•Respiratory and functional capacities were stable or improved in most patients.•Postinfusion, avalglucosidase alfa in plasma fell monoexponentially (t1/2z ∼1.0 h).•AUC for avalglucosidase alfa in plasma was 5–6 × higher for the 20 vs 5 mg/kg group.•Safety/exploratory efficacy data support further avalglucosidase alfa development.