alglucosidase alfa (Rx)

Brand and Other Names:Lumizyme, Myozyme

Dosing & Uses


Dosage Forms & Strengths

powder for injectable solution

  • 50mg/vial (Lumizyme)

Pompe Disease (GAA Deficiency)

20 mg/kg IV q2wk; infuse over ~4 hr  

Infusion rate: 1 mg/kg/hr IV initially, may increase by 2 mg/kg/hr q30min, not to exceed 7 mg/kg/hr

Dosage Forms & Strengths

powder for injectable solution

  • 5mg/vial (Myozyme)
  • 50mg/vial (Lumizyme)

Pompe Disease (GAA Deficiency)

20 mg/kg IV q2wk; infuse over ~4 hr  

Infusion rate: 1 mg/kg/hr IV initially, may increase by 2 mg/kg/hr q30min, not to exceed 7 mg/kg/hr


Adverse Effects


Pyrexia (92%)

Diarrhea (62%)

Rash (54%)

Infusion reactions (51%)

Vomiting (49%)

Cough (46%)

Diaper dermatitis (46%)

Pneumonia (46%)

Otitis media (44%)

URI (44%)

O2 saturation decreased (41%)

Gastroenteritis (41%)

Pharyngitis (36%)

Respiratory distress (33%)

Respiratory failure (31%)

Anemia (31%)

Oral candidiasis (31%)

Rhinorrhea (28%)

Catheter-related infection (28%)

GERD (26%)

Post-procedural pain (26%)

Bronchiolitis (23%)

Constipation (23%)

Nasopharyngitis (23%)

Tachycardia (23%)

Tachypnea (23%)

Bradycardia (21%)

Flushing (21%)

Urticaria (21%)

Frequency Not Defined


Blurred vision

Abdominal pain




Facial Erythema







Coronary artery disease



Postmarketing Reports

Anaphylactic shock, respiratory failure, respiratory arrest, cardiac arrest, hypoxia, dyspnea, wheezing, convulsions, peripheral coldness, restlessness, nervousness, back pain, stridor, pharyngeal edema, abdominal pain, apnea, muscle spasm, conjunctivitis, hyperparathyroidism

Proteinuria and nephrotic syndrome secondary to membranous glomerulonephritis, and necrotizing skin lesions reported



Black Box Warnings

Anaphylaxis and severe hypersensitivity

  • Life-threatening anaphylactic reactions and severe hypersensitivity reactions reported after IV infusions
  • Immune-mediated reactions presenting as proteinuria, nephrotic syndrome, and necrotizing skin lesions reported
  • Closely observe patients during and after administration and be prepared to manage anaphylaxis and hypersensitivity reactions
  • Inform patients and caregivers of the signs and symptoms of anaphylaxis, hypersensitivity reactions, and immune-mediated reactions and have them seek immediate medical care should signs and symptoms occur

Compromised cardiac or respiratory function

  • Infantile-onset Pompe disease patients with compromised cardiac or respiratory function may be at risk of serious acute exacerbation of their cardiac or respiratory compromise due to fluid overload
  • Additional is monitoring required in these patients




Anaphylaxis and hypersensitivity reactions have been observed in patients during and up to 3 hr after infusion; some of the 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 Immune-mediated cutaneous reactions have been reported including necrotizing skin lesions; these reactions occurred several weeks to 3 yr after initiation

Severe reactions are managed with infusion interruption, administration of antihistamines, corticosteroids, intravenous fluids, and/or oxygen, when clinically indicated; epinephrine may be administered; appropriate medical support, including cardiopulmonary resuscitation equipment, should be readily available when alglucosidase alfa is administered

Consider risks and benefits of re-administering alglucosidase alfa following anaphylactic or hypersensitivity reaction; monitor closely with appropriate resuscitation measures available, if decision is made to re-administer product

Nephrotic syndrome secondary to membranous glomerulonephritis was observed in some Pompe disease patients treated with alglucosidase alfa who had persistently positive anti-rhGAA IgG antibody titers; patients receiving alglucosidase alfa should undergo periodic urinalysis

Patients with acute underlying respiratory illness or compromised cardiac and/or respiratory function may be at risk of serious exacerbation of their cardiac or respiratory compromise during infusions

General anesthesia can be complicated by the presence of severe cardiac and skeletal (including respiratory) muscle weakness; extreme caution should be used when administering general anesthesia; ventricular arrhythmias and bradycardia, resulting in cardiac arrest or death, or requiring cardiac resuscitation or defibrillation have been observed in patients with infantile-onset Pompe disease with cardiac hypertrophy during general anesthesia for central venous catheter placement

As with all therapeutic proteins, there is potential for immunogenicity; monitor patients for IgG antibody formation every 3 months for 2 years and annually thereafter; consider testing for IgG titers if patients develop hypersensitivity reactions, other immune-mediated reactions, or lose clinical response; may also test patients who experience reduced clinical response for inhibitory antibody activity; patients who experience anaphylactic or hypersensitivity reactions may also be tested for IgE antibodies to alglucosidase alfa and other mediators of anaphylaxis

Immune Mediated Reactions

  • Immune tolerance induction administered in conjunction with may also aid tolerability of alglucosidase alfa under management of a clinical specialist knowledgeable in immune tolerance induction in pediatric Pompe disease to optimize treatment
  • Immune tolerance induction administered prior to and in conjunction with initiation of alglucosidase alfa reported to aid tolerability of alglucosidase alfa in CRIM-negative patients; CRIM status has been shown to be associated with immunogenicity and patients’ responses to enzyme replacement therapies
  • CRIM-negative infants with infantile-onset Pompe disease treated with alglucosidase alfa have shown poorer clinical response in the presence of high sustained IgG antibody titers and positive inhibitory antibodies compared to CRIM-positive infants

Pregnancy & Lactation


Data from postmarketing reports and published case reports with alglucosidase alfa use in pregnant women have not identified an associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes; the continuation of treatment during pregnancy should be individualized to pregnant woman; untreated disease may result in worsening disease symptoms in pregnant women

Pregnant women and women of reproductive potential should be encouraged to enroll in the Pompe patient registry; the registry will monitor effect of therapy on pregnant women and their offspring; for more information, visit or call 1-800-745-4447, extension 15500

Disease-associated maternal and/or embryo-fetal risk; untreated Pompe disease has been associated with worsening respiratory and musculoskeletal symptoms in some pregnant women

Animal data

  • 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


Available published literature suggests presence of alglucosidase alfa in human milk; there are no reports of adverse effects on the breastfed infant; there is no information on effects of alglucosidase alfa on milk production; developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for therapy and any potential adverse effects on breastfed child from drug or from underlying maternal condition

Lactating women with Pompe disease receiving treatment should be encouraged to enroll in Pompe disease registry

A lactating woman may consider interrupting breastfeeding, pumping and discarding breast milk during treatment and for 24 hours after therapy administration in order to minimize drug exposure to a breastfed infant

Pregnancy Categories

A: Generally acceptable. Controlled studies in pregnant women show no evidence of fetal risk.

B: May be acceptable. Either animal studies show no risk but human studies not available or animal studies showed minor risks and human studies done and showed no risk.

C: Use with caution if benefits outweigh risks. Animal studies show risk and human studies not available or neither animal nor human studies done.

D: Use in LIFE-THREATENING emergencies when no safer drug available. Positive evidence of human fetal risk.

X: Do not use in pregnancy. Risks involved outweigh potential benefits. Safer alternatives exist.

NA: Information not available.



Mechanism of Action

Recombinant acid alpha-glucosidase (GAA)

Carbohydrate groups on GAA molecule bind to mannose-6-phosphate receptors, then GAA is transported into cell where it undergoes proteolytic cleavage resulting in increased enzymatic glycogen cleavage


Half-Life: 2-3 hr

Peak Plasma Concentration: 162±31 mcg/m

AUC: 811±141 mcg-hr/mL

Vd: 80-110 mL/kg

Clearance: 20-30 mL/hr/kg



IV Incompatibilities

Do not mix with other drugs in same infusion line

IV Preparation

Do not use filter needles during preparation

Determine number of vials required for dose (20 mg/kg)

Remove the required number of vials from the refrigerator and allow them to reach room temperature prior to reconstitution (approximately 30 minutes)

Reconstitute each vial by slowly injecting 10.3 mL sterile water for injection

Avoid foaming & forceful addition of fluid

Protect reconstituted solution from light

Discard if discoloration or particulate matter present

Dilute (slowly) reconstituted solution in 0.9% NaCl to final concentration of 0.5-4 mg/mL

Filter diluted solution through 0.2 micron low protein-binding, in-line filter during administration

IV Administration

Administer by IV infusion over ~4 hr

Infuse with an inline protein binding 0.2-micron filter

1 mg/kg/hr IV infusion initially, may increase by 2 mg/kg/hr q30 min; not to exceed 7 mg/kg/hr

Do not infuse in same IV line with other products

Recommended infusion volume:

  • 1.25-2.5 kg: 25 mL
  • 2.6-10 kg: 50 mL
  • 10.1-20 kg: 100 mL
  • 20.1-30 kg: 150 mL
  • 30.1-35 kg: 200 mL
  • 35.1-50 kg: 250 mL
  • 50.1-60 kg: 300 mL
  • 60.1-100 kg: 500 mL
  • 100.1-120 kg: 600 mL
  • 120.1-140 kg: 700 mL
  • 140.1-160 kg: 800 mL
  • 160.1-180 kg: 900 mL
  • 180.1-200 kg: 1000 mL


Unreconstituted vials: Store at 2-8°C (36-46°F)

Protect from freezing

Do not shake

Reconstituted/diluted solution stable up to 24 hr at 2-8°C (36-46°F)

Protect from light



No images available for this drug.

Patient Handout

Patient Education
alglucosidase alfa intravenous


(AL-gloo-KOE-si-dase AL-fa)


WARNING: Alglucosidase alfa may cause serious (sometimes fatal) allergic reactions that may happen during and up to 3 hours after the infusion. A health care professional will monitor you closely during and after your infusion. Get medical help right away if you notice any signs of a serious allergic reaction, such as rash, itching/swelling (especially of the face/tongue/throat), severe dizziness, trouble breathing, or chest pain. Your doctor may prescribe other medications (such as antihistamines, corticosteroids) to treat these side effects. Your infusion may also be slowed down or stopped depending on your symptoms.Rarely, people using this medication have developed severe skin/kidney problems. Tell your doctor right away if you develop changes in skin color, change in the amount of urine, or pink/bloody urine.Before starting treatment with this medication, tell your doctor if you have a history of heart problems (such as heart failure), breathing problems (such as fluid in the lungs), or swelling. These problems may get worse during the infusion. Your doctor or nurse may monitor you for longer periods after your infusion.

USES: This medication is used by people with a certain inherited condition (Pompe disease) to reduce high levels of a certain substance (glycogen) that builds up in the blood. Having high levels of glycogen in the blood may cause muscle weakness, heart problems, and breathing problems. Alglucosidase alfa works by breaking down glycogen in the blood.

HOW TO USE: Read the Patient Information Leaflet if available from your pharmacist before you start using alglucosidase alfa and each time you get an infusion. If you have any questions, ask your doctor or pharmacist.This medication is given by injection into a vein by a health care professional. It is given as directed by your doctor, usually once every 2 weeks. It is injected slowly into a vein over 4 hours. The dosage is based on your medical condition, weight, and response to treatment.Infusion reactions may happen while you are receiving alglucosidase alfa and for a short time after. Tell your doctor or nurse right away if you have any symptoms of infusion reactions such as shortness of breath, fever, sweating, flushing, nausea, or vomiting. Your doctor may slow down or stop your treatment for some time if you get serious side effects. Your doctor may prescribe other medications for you to take to help decrease these side effects. Carefully follow your doctor's directions for all of your medications. See also Warning section.Use this medication regularly to get the most benefit from it. To help you remember, mark the days on the calendar when you need to receive the medication.

SIDE EFFECTS: See also Warning section.Remember that this medication has been prescribed because your doctor has judged that the benefit to you is greater than the risk of side effects. Many people using this medication do not have serious side effects.Alglucosidase alfa can commonly cause a rash that is usually not serious. However, you may not be able to tell it apart from a rare rash that could be a sign of a severe reaction. Get medical help right away if you develop any rash.This is not a complete list of possible side effects. If you notice other effects not listed above, contact your doctor or pharmacist.In the US -Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088 or at Canada - Call your doctor for medical advice about side effects. You may report side effects to Health Canada at 1-866-234-2345.

PRECAUTIONS: See also Warning section.Before using alglucosidase alfa, tell your doctor or pharmacist if you are allergic to it; or if you have any other allergies. This product may contain inactive ingredients which can cause allergic reactions or other problems. Talk to your pharmacist for more details.Before using this medication, tell your doctor or pharmacist your medical history.This drug may make you dizzy. Alcohol or marijuana (cannabis) can make you more dizzy. Do not drive, use machinery, or do anything that needs alertness until you can do it safely. Limit alcoholic beverages. Talk to your doctor if you are using marijuana (cannabis).Before having surgery, tell your doctor or dentist about all the products you use (including prescription drugs, nonprescription drugs, and herbal products).During pregnancy, this medication should be used only when clearly needed. Discuss the risks and benefits with your doctor.This medication passes into breast milk. Consult your doctor before breast-feeding.

DRUG INTERACTIONS: Drug interactions may change how your medications work or increase your risk for serious side effects. This document does not contain all possible drug interactions. Keep a list of all the products you use (including prescription/nonprescription drugs and herbal products) and share it with your doctor and pharmacist. Do not start, stop, or change the dosage of any medicines without your doctor's approval.

OVERDOSE: If someone has overdosed and has serious symptoms such as passing out or trouble breathing, call 911. Otherwise, call a poison control center right away. US residents can call their local poison control center at 1-800-222-1222. Canada residents can call a provincial poison control center.

NOTES: Lab and/or medical tests (such as urine tests, immunoglobulin levels) should be done while you are using this medication. Keep all medical and lab appointments.

MISSED DOSE: It is important to get each dose of this medication as scheduled. If you miss a dose, ask your doctor or pharmacist right away for a new dosing schedule.

STORAGE: Not applicable. This medication is given in a clinic or doctor's office and will not be stored at home.

Information last revised February 2023. Copyright(c) 2023 First Databank, Inc.

IMPORTANT: HOW TO USE THIS INFORMATION: This is a summary and does NOT have all possible information about this product. This information does not assure that this product is safe, effective, or appropriate for you. This information is not individual medical advice and does not substitute for the advice of your health care professional. Always ask your health care professional for complete information about this product and your specific health needs.



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Tier Description
1 This drug is available at the lowest co-pay. Most commonly, these are generic drugs.
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Code Definition
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