Substance / Medication

Alglucosidase alfa

Overview

Active Ingredient

alglucosidase alfa

RxNorm CUI

629565

Indications

® LUMIZYMEis a hydrolytic lysosomal glycogen-specific enzyme indicated for patients with Pompe disease (acid α-glucosidase [GAA] deficiency).

Labeler: Genzyme CorporationUpdated: 2025-01-10T00:00:00.000ZFull label on DailyMed

Contraindications

When this intervention should not be used

None.

Indications & Related Conditions

Conditions associated via SNOMED clinical relationships

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:

Survival and long-term outcomes in late-onset Pompe disease following alglucosidase alfa treatment: a systematic review and meta-analysis.

Schoser Benedikt, Stewart Andrew, Kanters Steve et al. · J Neurol · 2017

PMID: 27372449Meta-Analysis

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)

Switching Enzyme Replacement Therapy for Late-Onset Pompe Disease From Alglucosidase Alfa to Cipaglucosidase Alfa Plus Miglustat: Post Hoc Effect Size Analysis of PROPEL.

Kushlaf Hani, Díaz-Manera Jordi, Bratkovic Drago et al. · Muscle Nerve · 2025

PMID: 40342075RCT

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)

Analysis of the Italian cohort of late-onset Pompe disease (LOPD) patients after 10 and 15 years of therapy with alglucosidase alfa.

Mongini T, Gadaleta G, Alonge P et al. · J Neurol · 2025

PMID: 40643754Observational

Changes in forced vital capacity over ≤ 13 years among patients with late-onset Pompe disease treated with alglucosidase alfa: new modeling of real-world data from the Pompe Registry.

Berger Kenneth I, Chien Yin-Hsiu, Dubrovsky Alberto et al. · J Neurol · 2024

PMID: 38896264ObservationalFull text (PMC)

Real-world data of in-hospital administration of alglucosidase alfa in French patients with Pompe disease: results from the National Claims Database.

Attarian Shahram, Campana Emmanuelle Salort, Perrier Stanislas et al. · J Neurol · 2024

PMID: 38963441Observational

Effect of alglucosidase alfa dosage on survival and walking ability in patients with classic infantile Pompe disease: a multicentre observational cohort study from the European Pompe Consortium.

Ditters Imke Anne Maartje, Huidekoper Hidde Harmen, Kruijshaar Michelle Elisabeth et al. · Lancet Child Adolesc Health · 2022

PMID: 34822769Observational

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: Alglucosidase alfa (substance)
SNOMED CT: 703393008
UMLS CUI: C1695579
RxNorm CUI: 629565
Labeler: Genzyme Corporation

Clinical Data

This intervention maps to 1 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.