An Indirect Treatment Comparison of Avalglucosidase Alfa versus Cipaglucosidase Alfa Plus Miglustat in Patients with Late-Onset Pompe Disease.

ABSTRACT:

Introduction

No head-to-head studies comparing the efficacy of avalglucosidase alfa (AVA) with cipaglucosidase alfa + miglustat (Cipa+mig) have been conducted in patients with late-onset Pompe disease (LOPD). Two indirect treatment comparisons (ITCs) were conducted to estimate the effects of AVA versus Cipa+mig.

Methods

ITCs were conducted using simulated treatment comparisons (STCs), adjusting for differences in prognostic factors and treatment effect modifiers. An analysis of patients who were naïve to enzyme replacement therapy (ERT-naive) used anchored STC with individual patient data (IPD) from the Phase 3 COMET (NCT02782741) study of AVA versus alglucosidase alfa (ALG) and aggregate data from patients who were ERT-naïve in the Phase 3 PROPEL (NCT03729362) study of Cipa

SUBMITTER: Roberts ME 

PROVIDER: S-EPMC12579690 | biostudies-literature | 2025 Nov

REPOSITORIES: biostudies-literature