NEXVIAZYME (avalglucosidase alfa-ngpt)
NEXVIAZYME is indicated for the treatment of Pompe Disease.
How NEXVIAZYME Works
Avalglucosidase alfa-ngpt provides an exogenous source of the acid α-glucosidase (GAA) enzyme, which is deficient in patients with Pompe disease. The drug utilizes mannose-6-phosphate (M6P) to bind to receptors on the cell surface, facilitating its internalization and transport into lysosomes. Once inside the lysosome, the drug undergoes proteolytic cleavage to increase its enzymatic activity. This active enzyme then cleaves and degrades accumulated glycogen into glucose, addressing the intralysosomal accumulation of glycogen.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2021-08-06
- Revenue
- $224M (Q4-2025)
- Routes
- INJECTION
- Dosage Forms
- INJECTABLE
NEXVIAZYME Approval History
What NEXVIAZYME Treats
1 indicationsNEXVIAZYME is approved for 1 conditions since its original approval in 2021. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Pompe Disease
NEXVIAZYME Boxed Warning
SEVERE HYPERSENSITIVITY REACTIONS, INFUSION-ASSOCIATED REACTIONS, and RISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS WARNING: SEVERE HYPERSENSITIVITY REACTIONS, INFUSION-ASSOCIATED REACTIONS, and RISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS See full prescribing information for complete boxed warning. Hypersensitivity Reactions Including Anaphylaxis Appropriate medical monitoring and support measures, including cardiopulmonary resuscitation equipment, should be...
WARNING: SEVERE HYPERSENSITIVITY REACTIONS, INFUSION-ASSOCIATED REACTIONS, and RISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS WARNING: SEVERE HYPERSENSITIVITY REACTIONS, INFUSION-ASSOCIATED REACTIONS, and RISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS See full prescribing information for complete boxed warning. Hypersensitivity Reactions Including Anaphylaxis Appropriate medical monitoring and support measures, including cardiopulmonary resuscitation equipment, should be readily available. If a severe hypersensitivity reaction occurs, discontinue NEXVIAZYME immediately and initiate appropriate medical treatment. ( 5.1 ) Infusion-Associated Reactions (IARs) If severe IARs occur, consider immediate discontinuation and initiation of appropriate medical treatment. ( 5.2 ) Risk of Acute Cardiorespiratory Failure in Susceptible Patients Patients susceptible to fluid volume overload, or those with acute underlying respiratory illness or compromised cardiac or respiratory function, may be at risk of serious exacerbation of their cardiac or respiratory status during NEXVIAZYME infusion. ( 5.3 ) Hypersensitivity Reactions Including Anaphylaxis Patients treated with NEXVIAZYME have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Appropriate medical monitoring and support measures, including cardiopulmonary resuscitation equipment, should be readily available during NEXVIAZYME administration. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue NEXVIAZYME immediately and initiate appropriate medical treatment. In patients with severe hypersensitivity reactions, a desensitization procedure to NEXVIAZYME may be considered [see Warnings and Precautions (5.1) ] . Infusion-Associated Reactions (IARs) Patients treated with NEXVIAZYME have experienced severe IARs. If severe IARs occur, consider immediate discontinuation of NEXVIAZYME, initiation of appropriate medical treatment, and the benefits
NEXVIAZYME Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
Direct competitors
Same target(s) AND same indication — head-to-head.
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
Drugs Similar to NEXVIAZYME
3 of 4FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
3 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT04910776 Baby-COMET | EFC14462 U1111-1246-6645, 2024-513859-33 | Ph 3 | active not recruiting | Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa |
| NCT06666413 | LPS18086 U1111-1295-2947 | Ph 4 | recruiting | China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD |
| NCT02032524 NEO-EXT results posted | LTS13769 U1111-1147-3439 | Ph 2 | completed | Avalglucosidase Alfa Extension Study |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
NEXVIAZYME FDA Label Details
Indications & Usage
FDA Label (PDF)NEXVIAZYME is indicated for the treatment of Pompe Disease.
WARNING: SEVERE HYPERSENSITIVITY REACTIONS, INFUSION-ASSOCIATED REACTIONS, and RISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS WARNING: SEVERE HYPERSENSITIVITY REACTIONS, INFUSION-ASSOCIATED REACTIONS, and RISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS See full presc...
Pro Intelligence Preview
Deep insights for NEXVIAZYME
Revenue Insights
- • Q4-2025: $224M
- • Historical trend analysis
Patent Timeline
- • Patent expiration dates
- • Generic/biosimilar risk
Trial Analysis
- • 3 total trials
- • Stage: Expansion
Competitive Landscape
- • 4 similar drugs
- • Same target/indication analysis
Full approval history • All patents • Revenue trends • Competitor analysis
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment