Alglucosidase Alfa

Pompe disease (acid maltase deficiency, glycogen storage disease type II, GSD II, glycogenosis type II) is an inherited disorder of glycogen metabolism caused by the absence or marked deficiency of the lysosomal enzyme GAA. Alglucosidase alfa provides an exogenous source of GAA.

• LUMIZYME ® is a hydrolytic lysosomal glycogen-specific enzyme indicated for patients with Pompe disease (acid α-glucosidase [GAA] deficiency). LUMIZYME ® is a hydrolytic lysosomal glycogen-specific enzyme indicated for patients with Pompe disease (GAA deficiency).

• None. None.contraindicated

Administration of LUMIZYME should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis. ( 2.1 ) Recommended dosage is 20 mg/per kg body weight administered every 2 weeks as an intravenous infusion. The initial infusion rate should be no more than 1 mg/kg/hour ( 2.2 ) Reconstitute and dilute LUMIZYME prior to use. ( 2.3 ) See full prescribing information for storage of the reconstituted and diluted product and administration instructions ( 2.4 , 2.5 ) 2.1 Recommendations prior to LUMIZYME Treatment Administration of LUMIZYME should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis [see Warnings and Precautions (5.1) ] . Initiate LUMIZYME in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment [see Warnings and Precautions (5.1) ] . Prior to LUMIZYME administration, consider pretreating with antihistamines, antipyretics, and/or corticosteroids [see Warnings and Precautions (5.1 , 5.2) ] . LUMIZYME must be reconstituted and diluted prior to use [see Dosage and Administration (2.3) ] . Appropriate medical monitoring and support measures, including cardiopulmonary resuscitation equipment, should be readily available during LUMIZYME administration [see Warnings and Precautions (5.1) ] . 2.2 Recommended Dosage and Administration The recommended dosage of LUMIZYME is 20 mg/kg body weight administered every 2 weeks as an intravenous infusion.

Infusion-Associated Reactions (IARs) : If an IAR occurs, decreasing the infusion rate, temporarily stopping the infusion, and/or administration of antihistamines and/or antipyretics may ameliorate the symptoms. ( 5.2 ) Risk of Cardiac Arrhythmia and Sudden Cardiac Death during General Anesthesia for Central Venous Catheter Placement : Caution should be used when administering general anesthesia for the placement of a central venous catheter intended for LUMIZYME infusion. ( 5.5 ) Risk of Developing Anti-alglucosidase Alfa Antibodies : Patients with IOPD should have a cross-reactive immunologic material (CRIM) assessment early in their disease course and be managed by a clinical specialist knowledgeable in immune tolerance induction in Pompe disease. ( 5.6 ) 5.1 Hypersensitivity Reactions Including Anaphylaxis Hypersensitivity Reactions Including Anaphylaxis Hypersensitivity reactions including anaphylaxis have been observed in patients during and up to 3 hours after a LUMIZYME infusion. Some of the hypersensitivity reactions were life-threatening and included anaphylactic shock, cardiac arrest, respiratory arrest, respiratory distress, hypoxia, apnea, dyspnea, bradycardia, tachycardia, bronchospasm, throat tightness, hypotension, angioedema (including tongue or lip swelling, periorbital edema, and face edema), and urticaria.

The following serious adverse reactions are described below and elsewhere in the labeling: Hypersensitivity Reactions Including Anaphylaxis [see Warnings and Precautions (5.1) ] The most frequently reported adverse reactions (≥5%) in clinical trials were hypersensitivity reactions and included: anaphylaxis, rash, pyrexia, flushing/feeling hot, urticaria, headache, hyperhidrosis, nausea, cough, decreased oxygen saturation, tachycardia, tachypnea, chest discomfort, dizziness, muscle twitching, agitation, cyanosis, erythema, hypertension/increased blood pressure, pallor, rigors, tremor, vomiting, fatigue, and myalgia. ( 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 clinical practice. In clinical trials, the most common adverse reactions (≥5%) following intravenous alglucosidase alfa treatment were hypersensitivity reactions, and included anaphylaxis, rash, pyrexia, flushing/feeling hot, urticaria, headache, hyperhidrosis, nausea, cough, decreased oxygen saturation, tachycardia, tachypnea, chest discomfort, dizziness, muscle twitching, agitation, cyanosis, erythema, hypertension/increased blood pressure, pallor, rigors, tremor, vomiting, fatigue, and myalgia.

8.1 Pregnancy Pregnancy Exposure Registry There is a pregnancy exposure registry that monitors pregnancy outcomes in women exposed to LUMIZYME during pregnancy. Pregnant women and women of reproductive potential should be encouraged to enroll in the Pompe patient registry. The registry will monitor the effect of LUMIZYME on pregnant women and their offspring. For more information, visit www.registrynxt.com or call 1-800-745-4447, extension 15500. Risk Summary Data from postmarketing reports and published case reports with alglucosidase alfa use in pregnant women have not identified a LUMIZYME-associated risk of major birth defects, miscarriage, or adverse maternal or fetal 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 ). Reproduction studies performed in mice and rabbits at doses resulting in exposures up to 0.4 or 0.5 times the human steady-state AUC (area under the plasma concentration-time curve), respectively, during the period of organogenesis revealed no evidence of effects on embryo-fetal development. In mice there was an increase in pup mortality during lactation at maternal exposures 0.4 times the human steady-state AUC (see Data ) .

Metabolism

The pharmacokinetics of alglucosidase alfa was evaluated in 13 patients with infantile-onset Pompe disease, aged 1 month to 7 months, who received 20 mg/kg (approximately as a 4-hour infusion) or 40 mg/kg (approximately as a 6.5-hour infusion) of alglucosidase alfa every 2 weeks. The measurement of alglucosidase alfa plasma concentration was based on an activity assay using an artificial substrate.

• May 24, 2010BLABLA125291Genzyme

1. 1Inappropriate Schedule Of Product Administration1938.2%
2. 2Pyrexia1667.1%
3. 3Off Label Use1436.1%
4. 4Dyspnoea1355.7%
5. 5Malaise1024.3%
6. 6Fatigue964.1%
7. 7Pneumonia954.0%
8. 8Fall944.0%
9. 9Urticaria853.6%
10. 10Infusion Related Reaction833.5%
11. 11Drug Specific Antibody Present823.5%
12. 12Rash813.4%
13. 13Pain783.3%
14. 14Weight Increased682.9%
15. 15Asthenia662.8%

The 10 most recently updated of 46 ClinicalTrials.gov registrations naming Alglucosidase 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
• Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
  Recruiting · Observational · 400 enrolled · Duke University
  NCT01665326updated 2026-04-08
• PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
  Recruiting · Phase 1 · Interventional · 10 enrolled · University of California, San Francisco
  NCT04532047updated 2026-03-17
• 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
• Developing a Management Approach for Patients With "Late-Onset" Pompe Disease
  Active not recruiting · Observational · 20 enrolled · Duke University
  NCT03694561updated 2025-09-22
• A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease
  Completed · Phase 3 · Interventional · 125 enrolled · Amicus Therapeutics
  NCT03729362updated 2025-09-11
• Expanded Access for ATB200/AT2221 for the Treatment of IOPD
  Available · Expanded access · Amicus Therapeutics
  NCT04327973updated 2025-09-10
• Drug-drug Interaction Study
  Completed · Phase 2 · Interventional · 25 enrolled · Amicus Therapeutics
  NCT01380743updated 2025-06-26
• A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)
  Active not recruiting · Observational · 16 enrolled · Sanofi
  NCT04848779updated 2025-05-13

Frequently asked questions

How does Alglucosidase Alfa work?

Pompe disease (acid maltase deficiency, glycogen storage disease type II, GSD II, glycogenosis type II) is an inherited disorder of glycogen metabolism caused by the absence or marked deficiency of the lysosomal enzyme GAA. Alglucosidase alfa provides an exogenous source of GAA.

What is Alglucosidase Alfa used for?

According to FDA labeling, Alglucosidase Alfa carries indications including: LUMIZYME ® is a hydrolytic lysosomal glycogen-specific enzyme indicated for patients with Pompe disease (acid α-glucosidase [GAA] deficiency). LUMIZYME ® is a hydrolytic lysosomal glycogen-specific enzyme indicated for patients with Pompe disease (GAA deficiency).. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Alglucosidase Alfa?

Alglucosidase Alfa is classified as Enzymes, Hydrolytic Lysosomal Glycogen-specific Enzyme, Increased Glycogenolysis.

What are the brand names for Alglucosidase Alfa?

Alglucosidase Alfa is marketed under brand names including Lumizyme.

What are the contraindications for Alglucosidase Alfa?

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