Avalglucosidase Alfa

Pompe disease (also known as glycogen storage disease type II, acid maltase deficiency, and glycogenosis type II) is an inherited disorder of glycogen metabolism caused by a deficiency of the lysosomal enzyme acid α-glucosidase (GAA) that degrades glycogen to glucose in the lysosome. GAA deficiency results in intralysosomal accumulation of glycogen in various tissues.

• 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).

• None. None.contraindicated

Consider administering antihistamines, antipyretics, and/or corticosteroids prior to NEXVIAZYME administration to reduce the risk of IARs. ( 2.1 ) NEXVIAZYME is administered as intravenous infusion. For patients weighing ( 2.2 ): ≥30 kg, the recommended dosage is 20 mg/kg (of actual body weight) every two weeks. <30 kg, the recommended dosage is 40 mg/kg (of actual body weight) every two weeks. See the full prescribing information for dosage modifications due to hypersensitivity reactions or IARs. ( 2.3 ) Must be reconstituted and diluted prior to use. ( 2.4 ) For instructions on storage and administration, see full prescribing information. ( 2.5 , 2.6 ) 2.1 Recommendations Prior to NEXVIAZYME Treatment Prior to NEXVIAZYME administration, consider pretreating with antihistamines, antipyretics, and/or corticosteroids [see Warnings and Precautions (5.1 , 5.2) ] . NEXVIAZYME must be reconstituted and diluted prior to use [see Dosage and Administration (2.4) ] . Appropriate medical monitoring and support measures, including cardiopulmonary resuscitation equipment, should be readily available during NEXVIAZYME administration. 2.2 Recommended Dosage and Administration NEXVIAZYME is administered as intravenous infusion. For patients weighing: 30 kg or more- the recommended dosage is 20 mg/kg (of actual body weight) every two weeks [see Dosage and Administration (2.6) ] Less than 30 kg- the recommended dosage is 40 mg/kg (of actual body weight) every two weeks [see Dosage and Administration (2.6) ] The initial recommended infusion rate is 1 mg/kg/hour.

See boxed warning . ( 5.1 , 5.2 , 5.3) 5.1 Hypersensitivity Reactions Including Anaphylaxis Life-threatening hypersensitivity reactions, including anaphylaxis, have been reported in NEXVIAZYME-treated patients. In NEXVIAZYME clinical studies, 67 (48%) NEXVIAZYME-treated patients experienced hypersensitivity reactions, including 6 (4%) patients who reported severe hypersensitivity reactions and 3 (2%) patients who experienced anaphylaxis; 2 (1%) patients who experienced anaphylaxis discontinued from the study. Some of the hypersensitivity reactions were IgE mediated. Symptoms of severe hypersensitivity reactions (e.g., anaphylaxis) included chest discomfort, erythema, generalized edema, hypotension, hypoxia, rash, respiratory distress, tongue edema, and urticaria. Prior to NEXVIAZYME administration, consider pretreating with antihistamines, antipyretics, and/or corticosteroids. 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. Consider the risks and benefits of re-administering NEXVIAZYME following severe hypersensitivity reactions (including anaphylaxis). Patients may be rechallenged using slower infusion rates at a dosage lower than the recommended dosage. In patients with severe hypersensitivity reaction, desensitization measures to NEXVIAZYME may be considered.

The following serious adverse reactions are discussed in greater detail in other sections of the labeling: Hypersensitivity Reactions Including Anaphylaxis [see Warnings and Precautions (5.1) ] Infusion-Associated Reactions (IARs) [see Warnings and Precautions (5.2) ] The most common adverse reactions (>5%) were headache, fatigue, diarrhea, nausea, arthralgia, dizziness, myalgia, pruritus, vomiting, dyspnea, erythema, paresthesia and urticaria. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Genzyme at 1-800-633-1610, option 1 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. Adverse Reactions from Clinical Trials in the Pompe Disease Population The pooled safety analysis from 4 clinical trials in the Pompe disease population (including Study 1) with a mean exposure of 26 months and up to 85 months of treatment included a total of 141 NEXVIAZYME-treated patients (118 adult and 23 pediatric patients [see Clinical Studies (14.1) ] . Adverse reactions were similar across both adult and pediatric populations. Serious adverse reactions reported in 2 or more NEXVIAZYME-treated patients were respiratory distress, chills, and pyrexia.

8.1 Pregnancy Risk Summary Available data from case reports of NEXVIAZYME use in pregnant women are insufficient to evaluate for a drug associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes. However, available data from postmarketing reports and published case reports on alglucosidase alfa (another hydrolytic lysosomal glycogen-specific enzyme replacement therapy) use in pregnant women have not identified a drug-associated risk of adverse pregnancy outcomes. The continuation of treatment for Pompe disease during pregnancy should be individualized to the pregnant woman. Untreated Pompe disease may result in worsening disease symptoms in pregnant women (see Clinical Considerations ) . Embryo-fetal toxicity studies performed in pregnant mice resulted in maternal toxicity related to an immunologic response (including an anaphylactoid response) and embryo-fetal loss at 17 times the human steady-state AUC at the recommended biweekly dose of 20 mg/kg for LOPD patients weighing ≥30 kg or 10 times the human steady-state AUC at the recommended biweekly dose of 40 mg/kg for LOPD patients weighing <30 kg. Avalglucosidase alfa-ngpt did not cross the placenta in mice, therefore, the adverse effects were likely related to the immunologic response in the mothers.

Metabolism

The avalglucosidase alfa-ngpt exposure increases in an approximately proportional manner with increasing doses over a range from 5 to 20 mg/kg (0.25 to 1 time the approved recommended dosage in LOPD patients weighing greater than or equal to 30 kg or 0.125 to 0.5 times the approved recommended dosage in LOPD patients weighing less than 30 kg). No accumulation was observed following every two weeks dosing.

• Aug 6, 2021BLABLA761194Genzyme Corp

1. 1Drug Specific Antibody Present13116%
2. 2Off Label Use8410.0%
3. 3Weight Increased495.8%
4. 4Weight Decreased384.5%
5. 5Inappropriate Schedule Of Product Administration354.2%
6. 6Dyspnoea313.7%
7. 7Pyrexia313.7%
8. 8Condition Aggravated303.6%
9. 9Fatigue303.6%
10. 10Illness253.0%
11. 11Headache232.7%
12. 12Pneumonia212.5%
13. 13Product Preparation Error212.5%
14. 14Cough202.4%
15. 15Covid-19202.4%

The 10 most recently updated of 10 ClinicalTrials.gov registrations naming Avalglucosidase Alfa as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• Pompe Pregnancy Sub-Registry
  Recruiting · Observational · 20 enrolled · Genzyme, a Sanofi Company
  NCT00567073updated 2026-04-16
• Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa
  Active not recruiting · Phase 3 · Interventional · 17 enrolled · Sanofi
  NCT04910776updated 2026-04-08
• China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD
  Recruiting · Phase 4 · Interventional · 13 enrolled · Genzyme, a Sanofi Company
  NCT06666413updated 2026-03-23
• A Global Prospective Observational Registry of Patients With Pompe Disease
  Recruiting · Observational · 500 enrolled · Amicus Therapeutics
  NCT06121011updated 2026-03-10
• 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 · Phase 2 · Interventional · 22 enrolled · Genzyme, a Sanofi Company
  NCT03019406updated 2026-01-30
• Avalglucosidase Alfa Pregnancy Study
  Recruiting · Observational · 100 enrolled · Sanofi
  NCT05734521updated 2026-01-16
• Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)
  Active not recruiting · Phase 4 · Interventional · 17 enrolled · Genzyme, a Sanofi Company
  NCT05164055updated 2025-10-31
• 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 · Phase 3 · Interventional · 101 enrolled · Genzyme, a Sanofi Company
  NCT02782741updated 2024-04-04
• Avalglucosidase Alfa Extension Study
  Completed · Phase 2 · Interventional · 19 enrolled · Genzyme, a Sanofi Company
  NCT02032524updated 2024-03-01
• Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.
  Completed · Phase 1 · Interventional · 24 enrolled · Genzyme, a Sanofi Company
  NCT01898364updated 2023-03-23

Frequently asked questions

How does Avalglucosidase Alfa work?

Pompe disease (also known as glycogen storage disease type II, acid maltase deficiency, and glycogenosis type II) is an inherited disorder of glycogen metabolism caused by a deficiency of the lysosomal enzyme acid α-glucosidase (GAA) that degrades glycogen to glucose in the lysosome. GAA deficiency results in intralysosomal accumulation of glycogen in various tissues.

What is Avalglucosidase Alfa used for?

According to FDA labeling, Avalglucosidase Alfa carries indications including: 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).. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Avalglucosidase Alfa?

Avalglucosidase Alfa is classified as Enzymes, Hydrolytic Lysosomal Glycogen-specific Enzyme, Enzymatic Activity.

What are the brand names for Avalglucosidase Alfa?

Avalglucosidase Alfa is marketed under brand names including Nexviazyme.

What are the contraindications for Avalglucosidase Alfa?

Avalglucosidase Alfa labeling lists contraindications including: None. None.. Always consult the full prescribing information and a clinician.