Last reviewed 2026-05-19 · How we verify

NEXVIAZYME (AVALGLUCOSIDASE ALFA-NGPT)

NEXVIAZYME (generic name: AVALGLUCOSIDASE ALFA-NGPT) is a Hydrolytic Lysosomal Glycogen-specific Enzyme [EPC] drug developed by GENZYME CORP. It is currently FDA-approved for Late-onset Pompe disease, Pompe disease.

Nexviazyme is a hydrolytic enzyme used to treat Glycogen Storage Disease Type II (GSD-II) and Pompe Disease, including late-onset Pompe Disease. It works by breaking down glycogen through its mechanism as a glycogen hydrolytic enzyme.

At a glance

Approved indications

• Late-onset Pompe disease
• Pompe disease

Boxed warnings

• 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 and risks of readministering NEXVIAZYME following severe IARs. Patients with an acute underlying illness at the time of NEXVIAZYME infusion may be at greater risk for IARs. Patients with advanced Pompe disease may have compromised cardiac and respiratory function, which may predispose them to a higher risk of severe complications from IARs [see Warnings and Precautions (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 for whom fluid restriction is indicated may be at risk of serious exacerbation of their cardiac or respiratory status during NEXVIAZYME infusion. More frequent monitoring of vitals should be performed during NEXVIAZYME infusion in such patients [see Warnings and Precautions (5.3) ] .

Common side effects

• Headache
• Fatigue
• Diarrhea
• Nausea
• Arthralgia
• Dizziness
• Myalgia
• Pruritus
• Vomiting
• Dyspnea
• Erythema
• Paresthesia

Serious adverse events

• Hypersensitivity reactions (severe)
• Infusion-associated reactions (severe)
• Respiratory distress
• Chills
• Pyrexia
• Anaphylaxis
• Hypoxia
• Generalized edema
• Tongue edema
• Chest discomfort

Key clinical trials

• A Sub-registry to Observe the Effect of Alglucosidase Alfa or Avalglucosidase Alfa Treatment on Pregnancy and Infant Growth in Women With Pompe Disease (N/A)
• A Global Prospective Observational Registry of Patients With Pompe Disease (N/A)
• 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 Tre (Phase 2)
• A Descriptive Safety Study Based on Data Collected From Women and Their Offspring Exposed to Nexviazyme/Nexviadyme (Avalglucosidase Alfa-ngpt/Avalglucosidase Alfa) During Pregnancy and/or Lactation in (N/A)
• 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) (Phase 4)

Primary sources

Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.

Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

• NEXVIAZYME CI brief — competitive landscape report
• NEXVIAZYME updates RSS · CI watch RSS
• GENZYME CORP portfolio CI

Frequently asked questions about NEXVIAZYME

Related

• Drug class: All Hydrolytic Lysosomal Glycogen-specific Enzyme [EPC] drugs
• Manufacturer: GENZYME CORP — full pipeline
• Therapeutic area: All drugs in Other
• Indication: Drugs for Late-onset Pompe disease
• Indication: Drugs for Pompe disease

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing