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Drug

Alglucosidase alfa

Established medicine
ResearchOngoing clinical research
Also known as Aglucosidase alfa, Aglucosidase alpha, Lumizyme, Myozyme+3 more

Aglucosidase alfa, Aglucosidase alpha, Lumizyme, Myozyme, Alglucosidasa alfa, Alglucosidase alpha, Human acid precursor alpha-glucosidase, recombinant.

RxNorm629565ChEMBLCHEMBL1201824ATCA16AB07UNIIDTI67O9503
1
Active clinical trials
1
Regulatory events
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

An Open-label Ascending Dose Cohort Study to Assess the Safety, Pharmacokinetics, and Preliminary Efficacy of Avalglucosidase Alfa (NeoGAA, GZ402666) in Patients With Infantile-onset Pompe Disease Treated With Alglucosidase Alfa Who Demonstrate Clinical Decline or Sub-optimal Clinical Response

Clinical trial2026-01-30Results posted · ClinicalTrials.gov

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

Clinical trial2025-09-11Results posted · ClinicalTrials.gov

A Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatment

Clinical trial2024-07-25Completed · ClinicalTrials.gov

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

Clinical trial2024-04-04Results posted · ClinicalTrials.gov

A Long-term Study to Evaluate Growth and Development Outcomes in Patients With Infantile-Onset Pompe Disease Who Are Receiving Alglucosidase Alfa

Clinical trial2022-08-26Results posted · ClinicalTrials.gov

A Phase 3/4 Prospective Study to Characterize the Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease

Clinical trial2022-03-28Results posted · ClinicalTrials.gov

A Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment

Clinical trial2020-12-30Completed · ClinicalTrials.gov

Randomized, Double-Blind, Placebo-Controlled Study of the Safety, Efficacy and Pharmacokinetics of Myozyme in Patients With Late-Onset Pompe Disease.

Clinical trial2015-04-28Results posted · ClinicalTrials.gov

A Phase 4 Prospective Exploratory Muscle Biopsy, Biomarker, and Imaging Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa

Clinical trial2014-12-19Results posted · ClinicalTrials.gov

A Phase 3/4, Prospective, Multinational, Open-label, Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease

Clinical trial2014-12-01Terminated · ClinicalTrials.gov

A Phase 4 Open Label, Prospective Study in Patients With Pompe Disease to Evaluate The Efficacy and Safety of Alglucosidase Alfa Produced at the 4000L Scale

Clinical trial2014-12-01Terminated · ClinicalTrials.gov

Approval: Myozyme (EMA)

Regulatory2006-03-28EMA

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Alglucosidase alfa
Aliases & brands
Aglucosidase alfaAglucosidase alphaLumizymeMyozymeAlglucosidasa alfaAlglucosidase alphaHuman acid precursor alpha-glucosidase, recombinant
RxNorm CUI
629565
ChEMBL ID
CHEMBL1201824
ATC codes
A16AB07
UNII
DTI67O9503
Regulatory jurisdictions
ema

Regulatory timeline

1 event

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval
2006-03-28
Latest approval
2006-03-28
Authorities
EMA
Total events
1
emaEuropean Medicines Agency· 1 event
2006-03-28Approval
Approval: Myozyme (EMA)
Indication: Myozyme is indicated for long-term enzyme-replacement therapy (ERT) in patients with a confirmed diagnosis of Pompe disease (acid-?-glucosidase deficiency). In patients with… Show full indication

Myozyme is indicated for long-term enzyme-replacement therapy (ERT) in patients with a confirmed diagnosis of Pompe disease (acid-?-glucosidase deficiency). In patients with late-onset Pompe disease the evidence of efficacy is limited.

Evidence ↗

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

23 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
23
registered trials across all phases
LATEST COMPLETION 2024
1
Active studies
17
Late-stage (III+)
20
Completed
2
Discontinued
PHASE DISTRIBUTIONn = 23
Phase 1 / 21Phase 25Phase 2 / 32Phase 34Phase 410Phase N / A1

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

A Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatment

CompletedPH IVGenzyme, a Sanofi Companyn=412024

A Long-term Study to Evaluate Growth and Development Outcomes in Patients With Infantile-Onset Pompe Disease Who Are Receiving Alglucosidase Alfa

CompletedPH IVGenzyme, a Sanofi Companyn=122021

A Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment

CompletedPH IVGenzyme, a Sanofi Companyn=102020

A Phase 3/4 Prospective Study to Characterize the Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease

CompletedPH IVGenzyme, a Sanofi Companyn=212020

A Phase 4 Open Label, Prospective Study in Patients With Pompe Disease to Evaluate The Efficacy and Safety of Alglucosidase Alfa Produced at the 4000L Scale

TerminatedPH IVGenzyme, a Sanofi Companyn=1132014

Recent completions

Trials that read out recently, adding to the completed evidence base.

A Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatment

CompletedPH IVGenzyme, a Sanofi Companyn=412024

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

CompletedPH IIIGenzyme, a Sanofi Companyn=1012023

A Long-term Study to Evaluate Growth and Development Outcomes in Patients With Infantile-Onset Pompe Disease Who Are Receiving Alglucosidase Alfa

CompletedPH IVGenzyme, a Sanofi Companyn=122021

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

CompletedPH IIIAmicus Therapeuticsn=1252021

A Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment

CompletedPH IVGenzyme, a Sanofi Companyn=102020

A Phase 3/4 Prospective Study to Characterize the Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease

CompletedPH IVGenzyme, a Sanofi Companyn=212020
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • ChEMBL (EMBL-EBI) & UniProt — pharmacology and targets
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.