NEXVIAZYME
NEXVIAZYME (avalglucosidase alfa-ngpt) is a hydrolytic lysosomal glycogen-specific enzyme indicated for the treatment of late-onset Pompe disease. This therapy is approved for use in adult and pediatric patients aged one year and older who have a deficiency in lysosomal acid alpha-glucosidase (GAA). By addressing this inherited disorder of glycogen metabolism, the drug serves as an exogenous enzyme source to manage the underlying cause of the 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.
Details
- Status
- Prescription
- First Approved
- 2021-08-06
- 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
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Trial Timeline
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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
ProIndications & Usage
FDA Label (PDF)NEXVIAZYME is indicated for the treatment of patients 1 year of age and older with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency). NEXVIAZYME is a hydrolytic lysosomal glycogen-specific enzyme indicated for the treatment of patients 1 year of age and older with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency).
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...
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Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.