ArticleAdvances in therapy2025
An Indirect Treatment Comparison of Avalglucosidase Alfa versus Cipaglucosidase Alfa Plus Miglustat in Patients with Late-Onset Pompe Disease.
Article in Advances in therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 5 registered trials, which are not on this map. Cited by 2 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
An Open-label, Multicenter, Multinational, Ascending Dose Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Repeated Biweekly Infusions of neoGAA in naïve and Alglucosidase Alfa Treated Late-onset Pompe Disease Patients.
An Open-label, Multicenter, Multinational Extension Study of the Long-term Safety and Pharmacokinetics of Repeated Biweekly Infusions of Avalglucosidase Alfa (neoGAA, GZ402666) in Patients With Pompe Disease
An Open-Label, Fixed-Sequence, Ascending-Dose, First-in-Human Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of Intravenous Infusions of ATB200 Co-Administered With Oral AT2221 in Adult Subjects With Pompe Disease
A Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Avalglucosidase Alfa (neoGAA, GZ402666) and Alglucosidase Alfa in Treatment naïve Patients With Late-onset Pompe Disease
A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease Compared With Alglucosidase Alfa/Placebo
Who cites it
2 citing papers in PubMed.
- Review
- Comprehensive review of recent advances in Pompe disease: pathogenesis, management, and future directions.Frontiers in neurologyReview
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
9 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
introductionNo 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.
methodsITCs 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+mig versus ALG + placebo. For patients who were ERT-experienced, an analysis used unanchored STC with IPD for AVA from the COMET open-label extension, and from NEO-1 (NCT01898364)/NEO-EXT (NCT02032524) studies, and aggregate Cipa+mig data from PROPEL and ATB200-02 (NCT02675465).
resultsIn patients who were ERT-naïve, the difference (95% confidence interval, CI) in mean change from baseline (CFB) at Weeks 49-52 in forced vital capacity percent predicted (FVCpp) showed a numerical improvement for AVA versus Cipa+mig with values of 5.49% (- 0.87, 11.86) with mixed model repeated measures analysis (MMRM/MMRM) and 4.69% (- 3.22, 12.61) with MMRM/analysis of covariance (ANCOVA). For the 6-min walk test (6MWT), differences were 57.08 m (11.04, 103.12) with MMRM/MMRM and 41.88 m (- 5.46, 89.22) with MMRM/ANCOVA, the former being statistically significant (p < 0.02) and the latter numerically in favour of AVA. In patients who were ERT-experienced, at Weeks 48-52 differences for AVA versus Cipa+mig with MMRM/MMRM (CIs/p values unavailable) were 1.40% for FVCpp and 18.85 m for 6MWT; with MMRM/Mean CFB, differences of 1.16% (- 1.88, 4.19) for FVCpp and 7.67 m (- 21.67, 37.02) for 6MWT, indicating a numerical improvement in favour of AVA.
conclusionsITCs suggest more favourable respiratory and mobility outcomes with AVA versus Cipa+mig in patients with LOPD, regardless of prior ERT-experience.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.