Drug updated on 11/5/2024
| Dosage Form | Injection (intravenous; 100 mg) |
| Drug Class | Hydrolytic lysosomal glycogen-specific enzymes |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the treatment of patients 1 year of age and older with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency).
Latest News
Summary
- This summary is based on the review of three randomized controlled trial(s). [1-3]
- Avalglucosidase alfa demonstrated superior effectiveness over alglucosidase alfa in improving health-related quality of life (HRQoL) in treatment-naive patients with late-onset Pompe disease (LOPD), with significant improvements observed in domains such as shortness of breath, fatigue, and overall activity levels (e.g., PDSS, EQ-5D-5L, and PGIC scores).
- In terms of respiratory function and physical endurance, avalglucosidase alfa led to a greater increase in forced vital capacity (FVC) percent predicted (2.89% vs. 0.46%) and 6-minute walk test distance (by 30.01 meters) compared to alglucosidase alfa over 49 weeks, with these improvements maintained or enhanced during the extension phase.
- Avalglucosidase alfa's overall effectiveness, including significant improvements in HRQoL and functional outcomes, positions it as a more favorable treatment option compared to alglucosidase alfa for LOPD patients.
- Treatment-emergent adverse events (TEAEs) were reported in 45% of patients in the avalglucosidase alfa group and 49% in the alglucosidase alfa group over 49 weeks, with infusion-associated reactions occurring in 26% and 33% of patients, respectively. Serious TEAEs were reported in 16% of avalglucosidase alfa patients compared to 25% in the alglucosidase alfa group.
- Antidrug antibody responses were observed in both groups, with high and persistent titers more common in the alglucosidase alfa group (33%) compared to the avalglucosidase alfa group (20%). No new safety concerns were identified with avalglucosidase alfa.
- The population studied primarily included treatment-naive patients with late-onset Pompe disease (LOPD), ranging from 3 years of age and older. The safety and effectiveness outcomes, including improvements in respiratory function and endurance, are relevant to this broad patient population. No specific subgroup analyses or significant differences in effectiveness among different population types or subgroups were reported.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Nexviazyme (avalglucosidase alfa-ngpt) Prescribing Information. | 2023 | Genzyme Corporation Cambridge, MA |
Randomized Controlled Trials
| Document Title | Sex Distribution | Year | Source |
|---|---|---|---|
| Effect of avalglucosidase alfa on disease-specific and general patient-reported outcomes in treatment-naive adults with late-onset Pompe disease compared with alglucosidase alfa: Meaningful change analyses from the Phase 3 COMET trial | 100Subjects F: 48% M: 52% | 2024 | Molecular Genetics and Metabolism |
| Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial | 100Subjects F: 48% M: 52% | 2023 | JAMA Neurology |
| Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial | 100Subjects F: 48% M: 52% | 2021 | The Lancet |
Sex Distribution:
F:48%
M:52%
100Subjects
Year:
2024
Source:Molecular Genetics and Metabolism
Sex Distribution:
F:48%
M:52%
100Subjects
Year:
2023
Source:JAMA Neurology
Sex Distribution:
F:48%
M:52%
100Subjects
Year:
2021
Source:The Lancet