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Drug

Avalglucosidase alfa

Approved medicine
ResearchOngoing clinical research

Also known as NeoGAA, Nexviazyme, Neo-recombinant human acid alpha-glucosidase.

RxNorm2565814ChEMBLCHEMBL4594320ATCA16AB22UNIIEO144CP0X9
5
Active clinical trials
1
Regulatory events
Latest activity
beta

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

An Open Label, Single Center, Exploratory Study of the Safety and Efficacy of Avalglucosidase Alfa Patients With Non-classic Pompe Disease Aged ≥ 5 Years

Clinical trial2026-06-17Results expected Q1 2028 · ClinicalTrials.gov

A Single-arm, Open-label, Phase 4 Study to Evaluate the Safety and Efficacy of Avalglucosidase Alfa in Chinese Participants With Infantile-onset Pompe Disease (IOPD)

Clinical trial2026-03-23Results expected Q2 2028 · ClinicalTrials.gov

An Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Avalglucosidase Alfa in Treatment naïve Pediatric Participants With Infantile-Onset Pompe Disease (IOPD)

Clinical trial2026-03-02Primary completion · ClinicalTrials.gov

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 French Multicenter Open Label Phase 4 Extension Study of Long-term Safety and Efficacy in Patients With Pompe Disease Who Previously Participated in Avalglucosidase Alfa Development Studies in France

Clinical trial2025-10-31Results expected Q3 2026 · 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

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

Clinical trial2024-03-01Results posted · ClinicalTrials.gov

Approval: Nexviadyme (EMA)

Regulatory2022-06-24EMA

Profile

Identifiers & mechanism

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

Canonical name
Avalglucosidase alfa
Aliases & brands
NeoGAANexviazymeNeo-recombinant human acid alpha-glucosidase
RxNorm CUI
2565814
ChEMBL ID
CHEMBL4594320
ATC codes
A16AB22
UNII
EO144CP0X9
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
2022-06-24
Latest approval
2022-06-24
Authorities
EMA
Total events
1
emaEuropean Medicines Agency· 1 event
2022-06-24Approval
Approval: Nexviadyme (EMA)

Indication: Nexviadyme (avalglucosidase alfa) is indicated for long-term enzyme replacement therapy for the treatment of patients with Pompe disease (acid ?-glucosidase deficiency).

Evidence ↗

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

Clinical trials

7 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
7
registered trials across all phases
LATEST COMPLETION 2023
5
Active studies
1
Recruiting
5
Late-stage (III+)
2
Completed
PHASE DISTRIBUTIONn = 7
Phase 22Phase 32Phase 43

Late-stage studies

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

A Single-arm, Open-label, Phase 4 Study to Evaluate the Safety and Efficacy of Avalglucosidase Alfa in Chinese Participants With Infantile-onset Pompe Disease (IOPD)

RecruitingPH IVGenzyme, a Sanofi Companyn=132028

An Open Label, Single Center, Exploratory Study of the Safety and Efficacy of Avalglucosidase Alfa Patients With Non-classic Pompe Disease Aged ≥ 5 Years

Active, not recruitingPH IVIris Plugn=62028

A French Multicenter Open Label Phase 4 Extension Study of Long-term Safety and Efficacy in Patients With Pompe Disease Who Previously Participated in Avalglucosidase Alfa Development Studies in France

Active, not recruitingPH IVGenzyme, a Sanofi Companyn=172026

An Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Avalglucosidase Alfa in Treatment naïve Pediatric Participants With Infantile-Onset Pompe Disease (IOPD)

Active, not recruitingPH IIISanofin=172027

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
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.