Avalglucosidase alfa

PreclinicalProtein / enzyme

Avalglucosidase alfa is a protein / enzyme that works as a Recombinant Acid Alpha-Glucosidase.[2] It is being investigated across 1 indication, with its most advanced program in Preclinical.[1]

Avalglucosidase alfa is one of 27 enzyme replacement programs targeting Pompe Disease. It is currently in Preclinical.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

Pompe DiseaseUnknown

Pompe DiseasePreclinical

Unknown

Competition

Competitive Landscape

38 competitors in Pompe Disease

#DrugCompanyPhaseTrials

Rituxan

Roche

Approved

3 2

Trexall

Alnylam Pharmaceuticals

Approved

2 3

Asceniv

CSL Behring

Approved

1 4

Velcade

SShilpa Grover, MD, MPH

Approved

1 5

Salbutamol

GSK

Approved

1 6

Opfolda

Johnson & Johnson

Phase 3

3 7

BMN 333

BioMarin Pharmaceutical

Phase 3

2 8

Clenbuterol

UUniversity of Copenhagen

Phase 2

2 9

Proair HFA

GGlaxo Dba Glaxosmithkline

Phase 1

2 10

Benadryl

PParkedale

Phase 1

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q3 2026

Post-ApprovalActive, not recruiting

Pompe Disease

NCT05164055

Q3 2027

Phase 3Active, not recruiting

Efficacy and Safety

NCT04910776

Phase 2Active, not recruiting

Pompe Disease

NCT03019406

Q2 2028

Post-ApprovalRecruiting

ART

NCT06666413

Q4 2032

Recruiting

Pregnancy

NCT05734521

Clinical

Trials by Indication[1]

Pompe Disease3 trials

Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)

Active, not recruiting· Sanofi logo

Sanofi·Phase 4·NCT05164055·17 enrolled

A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa

Active, not recruiting· Sanofi logo

Sanofi·Phase 2·NCT03019406·22 enrolled

Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease

Completed· Sanofi logo

Sanofi·Phase 3·NCT02782741·101 enrolled

Pregnancy1 trial

Avalglucosidase Alfa Pregnancy Study

Recruiting· Sanofi logo

Sanofi··NCT05734521·100 enrolled

Efficacy and Safety1 trial

Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa

Active, not recruiting· Sanofi logo

Sanofi·Phase 3·NCT04910776·17 enrolled

ART1 trial

China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD

Recruiting· Sanofi logo

Sanofi·Phase 4·NCT06666413·13 enrolled

Unspecified1 trial

Avalglucosidase Alfa Extension Study

Completed· Sanofi logo

Sanofi·Phase 2·NCT02032524·19 enrolled

Sources

References

[1]
ClinicalTrials.gov — 7 trial records for Avalglucosidase alfa. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Avalglucosidase alfa compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Avalglucosidase alfa. EMBL-EBI & Wellcome Sanger Institute.
[4]
PubMed — 1 publication indexed for Avalglucosidase alfa. U.S. National Library of Medicine.
[5]
SEC EDGAR — Sanofi company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: Recombinant Acid Alpha-Glucosidase[2]
Modality: Protein / enzyme[2]
Highest Phase: Preclinical[1]
Company: Sanofi

Key Publications[4]

Avalglucosidase Alfa vs Alglucosidase Alfa in Late-Onset Pompe: COMET

Lancet NeurolJul 14, 2022DOI: 10.1016/S1474-4422(22)00211-5

Avalglucosidase alfa

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q3 2026

Q3 2027

Q2 2028

Q4 2032

Trials by Indication[1]

Pompe Disease3 trials

Pregnancy1 trial

Efficacy and Safety1 trial

ART1 trial

Unspecified1 trial

References

Drug Info

Quick Facts

Key Publications[4]

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