TABLE 1.

Avalglucosidase in the literature. Notable studies comparing the efficacy of avalglucosidase alfa and alglucosidase alfa.

Study	Population, dosages	Results
Long-Term Safety and Efficacy of Avalglucosidase Alfa in Patients with Late-Onset Pompe Disease. Dimachkie, Mazen M., et al. (2022)	n = 7 treatment naïve, n = 10 switch patients (who received alglucosidase alfa for 0.9–7 years), all adults with LOPD. Doses of 5, 10, or 20 mg/kg q2w for 32–45 months and then 20 mg/kg q2w for all	Upright FVC and 6MWT distance remained stable in most participants, and improvements in 6MWT distance were observed in most participants <45 years at enrollment in both the naïve and switch groups
Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial. Diaz-Manera et al. (2021), Kishnani et al. (2023)	Patients ≥3 years of age with LOPD. n = 51 patients on avalglucosidase alfa, n = 49 on alglucosidase alfa in primary period. All on avalglucosidase alfa in extension. Doses of 20 mg/kg q2w	Outcomes at the primary endpoint of 49 weeks showed an increased mean improvement in percent predicted upright FVC on avalglucosidase alfa compared to alglucosidase alfa. This was sufficient to meet noninferiority criteria, but it narrowly missed the threshold for superiority (p = 0.063). Other findings include a greater increase in 6MWT distance on avalglucosidase alfa versus alglucosidase alfa. In the 49-week extension period, the results were consistent with the original findings demonstrating maintenance of positive clinical outcomes
Mini-Comet: Individual-Level Treatment Responses in Infantile-Onset Pompe Disease Participants Receiving Avalglucosidase Alfa or Alglucosidase Alfa Who Previously Received Alglucosidase Alfa. Kishnani, Priya, et al. (2021)	n = 22 patients with IOPD. Doses of 20 and 40 mg/kg q2w	Exploratory efficacy outcomes (6MWT, GMFM-88, QMFT, Pompe-PEDI, ptosis, and LVM z-score) improved or stabilized with avalglucosidase alfa at 40 mg/kg q2w, whereas these parameters stabilized or declined with 20 mg/kg q2w or on alglucosidase alfa