Efficacy and safety of avalglucosidase alfa in Japanese patients with late-onset and infantile-onset Pompe diseases: A case series from clinical trials.

ABSTRACT:

Background

The efficacy and safety of avalglucosidase alfa for Pompe disease (PD) have been demonstrated in a global Phase 3 trial (COMET) in patients with late-onset PD (LOPD) and a global Phase 2 trial (Mini-COMET) in patients with infantile-onset PD (IOPD). This case series examines the individual results of three Japanese patients enrolled in these trials.

Methods

Case reports were assembled from data collected in the COMET and Mini-COMET trials. Detailed methods have been reported previously. The primary endpoint of COMET was change from baseline to week 49 in upright forced vital capacity percent (FVC %) predicted. The primary endpoint of Mini-COMET was safety and tolerability of avalglucosidase alfa. In both trials, key secondary endpoints included motor function test

SUBMITTER: Mori-Yoshimura M 

PROVIDER: S-EPMC11743810 | biostudies-literature | 2025 Mar

REPOSITORIES: biostudies-literature