Substance / Medication

Avalglucosidase alfa

Overview

Active Ingredient

avalglucosidase alfa

RxNorm CUI

2565814

Indications

NEXVIAZYME is indicated for the treatment of patients 1 year of age and older with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency).

Labeler: Genzyme CorporationUpdated: 2025-06-05T00:00:00.000ZFull label on DailyMed

Contraindications

When this intervention should not be used

None.

Indications & Related Conditions

Conditions associated via SNOMED clinical relationships

Deficiency of maltase

Glycogen heart disease

Administration & Protocol

Dosing, route, and treatment protocol

Detailed dosage and administration information is available in the full FDA drug label.

View full prescribing information on DailyMed

Monitoring & Follow-Up

Biomarkers relevant to this intervention via related conditions

No monitoring biomarkers have been mapped yet. Biomarker-intervention linkages are derived through related conditions and will expand as the knowledge graph grows.

Research Evidence

Published studies and systematic reviews

Sort:

Efficacy and safety of avalglucosidase alfa in patients with late-onset Pompe disease after 145 weeks of treatment during the COMET trial.

Kishnani Priya S, Díaz-Manera Jordi, Illarioshkin Sergey et al. · J Neurol · 2025

PMID: 40817977RCTFull text (PMC)

Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial.

Kishnani Priya S, Diaz-Manera Jordi, Toscano Antonio et al. · JAMA Neurol · 2023

PMID: 37036722RCTFull text (PMC)

Safety and efficacy of avalglucosidase alfa in individuals with infantile-onset Pompe disease enrolled in the phase 2, open-label Mini-COMET study: The 6-month primary analysis report.

Kishnani Priya S, Kronn David, Brassier Anaïs et al. · Genet Med · 2023

PMID: 36542086RCT

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

Roberts Mark E, Proskorovsky Irina, Guyot Patricia et al. · Adv Ther · 2025

PMID: 40920287ObservationalFull text (PMC)

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 Jordi, Kishnani Priya S, Kushlaf Hani et al. · Lancet Neurol · 2021

PMID: 34800399Observational

Marked Improvements in Airway Abnormalities and Multifaceted Outcomes After 2 Years Switching to Avalglucosidase Alfa: Evaluation of A 19-Year-Old Male Diagnosed With Late-Onset Pompe Disease.

Lu Chih-Hsuan, Niu Dau-Ming, Yeou Yuh-Jing et al. · Am J Med Genet A · 2026

PMID: 40913330Case Report

Successful desensitization protocol to alglucosidase and avalglucosidase alfa in a patient with infantile-onset Pompe disease.

Gendive Miriam, Delgado Teresa C, Unceta María et al. · Mol Genet Metab Rep · 2025

PMID: 40206190Case ReportFull text (PMC)

Rapid Regression of Myocardial Hypertrophy with First-Line Avalglucosidase Alfa in Infantile-Onset Pompe Disease: A Case Report.

Uemura Natsumi, Fujino Mitsuhiro, Higuchi Shinji et al. · Pediatr Cardiol · 2026

PMID: 41511518Other

Efficacy of Switching Therapy From Alglucosidase Alfa to Avalglucosidase Alfa on Respiratory Function in Participants With Late-Onset Pompe Disease: A Post Hoc Analysis From the COMET Trial.

Kishnani Priya S, Boentert Matthias, Wenninger Stephan et al. · JIMD Rep · 2025

PMID: 40799512OtherFull text (PMC)

Efficacy of transitioning from alglucosidase alfa to avalglucosidase alfa in infantile-onset Pompe disease: A single-center cohort analysis.

Chien Yin-Hsuan, Chen Hui-An, Hsu Rai-Hseng et al. · Genet Med · 2025

PMID: 39927452Other

Research data from MEDLINE/PubMed

Benefits & Expected Outcomes

Benefits, expected outcomes, efficacy data, and NNT (Number Needed to Treat) are pending physician authorship and evidence review.

Risks & Side Effects

Adverse reaction and safety data for this drug is sourced from the FDA-approved label.

View adverse reactions & drug interactions on DailyMed

Related Symptoms

Symptoms associated with conditions this intervention addresses

No related symptoms have been mapped yet. Symptom linkages are derived through associated conditions.

Alternatives & Comparisons

Alternative treatments, comparison data, and clinical decision support are pending physician authorship.

Medical Disclaimer

This information is for educational purposes only and does not constitute medical advice, diagnosis, or treatment. Always consult with a qualified healthcare provider regarding any medical condition or treatment plan.

Do not start, stop, or change any treatment without consulting your healthcare provider.

Quick Facts

Type: Substance / Medication
Fully Specified Name: Avalglucosidase alfa (substance)
SNOMED CT: 1172659000
UMLS CUI: C5418318
RxNorm CUI: 2565814
Labeler: Genzyme Corporation

Clinical Data

This intervention maps to 2 entities in the Ltrl knowledge graph.

Conditions

Biomarkers

Specialists

Symptoms

Data is sourced from SNOMED CT, UMLS, and the Ltrl clinical knowledge graph. Content sections marked as pending require physician authorship. Consult a healthcare provider before starting any treatment.