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Alglucosidase Alfa

Medically reviewed by Last updated on Sep 26, 2020.


(al gloo KOSE i dase AL fa)

Index Terms

  • Alglucosidase
  • GAA
  • rhGAA

Dosage Forms

Excipient information presented when available (limited, particularly for generics); consult specific product labeling.

Solution Reconstituted, Intravenous [preservative free]:

Lumizyme: 50 mg (1 ea) [contains polysorbate 80]

Brand Names: U.S.

  • Lumizyme

Pharmacologic Category

  • Enzyme


Alglucosidase alfa is a recombinant form of the enzyme acid alpha-glucosidase (GAA), which is required for glycogen cleavage. Due to an inherited GAA deficiency or absence, glycogen accumulates in the tissues of patients with Pompe disease, leading to progressive muscle weakness. In infantile-onset Pompe disease, glycogen accumulates in cardiac and skeletal muscles and hepatic tissue, leading to cardiomyopathy and respiratory failure. Juvenile- and adult-onset Pompe disease are limited to glycogen accumulation in skeletal muscle, leading to respiratory failure. Alglucosidase alfa binds to mannose-6-phosphate receptors on the cell surface, is internalized, and transported to lysosomes where it is activated for increased enzymatic glycogen cleavage.


Vss: Infants 1 to 7 months: 96 ± 16 mL/kg

Half-Life Elimination

Infants 1 to 7 months: 2.3 hours; Adults: 2.4 hours

Special Populations Note

Antibody formation: Higher mean clearance (50%) was observed in patients who tested positive for antibodies to alglucosidase alfa.

Use: Labeled Indications

Pompe disease: For use in patients with Pompe disease (acid alpha-glucosidase [GAA] deficiency).


There are no contraindications listed in the US manufacturer's labeling.

Canadian labeling: Hypersensitivity to alglucosidase alfa or any component of the formulation.

Dosing: Adult

Pompe disease: Noninfantile, late-onset: IV: 20 mg/kg every 2 weeks

Dosing: Pediatric

Note: Patients with infantile-onset Pompe disease should have a cross-reactive immunological material (CRIM) assessment early in their disease course and be managed by a clinical specialist knowledgeable in immune tolerance induction.

Infantile-onset Pompe disease: Infants, Children, and Adolescents: IV: 20 mg/kg/dose every 2 weeks.

Late-onset (noninfantile) Pompe disease: Children and Adolescents: IV: 20 mg/kg/dose every 2 weeks.


Allow vials to reach room temperature prior to reconstitution (~30 minutes). Reconstitute each vial with 10.3 mL of SWFI. Inject slowly down internal side wall of vial (do not inject into powder; avoid foaming). Roll and tilt gently; do not invert, swirl, or shake. Resulting solution contains 5 mg/mL. To make final infusion, add the desired amount of reconstituted solution (based on patient weight) to 50 to 1000 mL of NS (do not use filter needle to prepare) to a final concentration of 0.5 to 4 mg/mL. Remove airspace from infusion bag prior to admixture to minimize particle formation due to sensitivity of drug to air-liquid interfaces. Gently invert or massage infusion bag to mix; do not shake.


IV: Infuse through a separate line via an inline, low-protein-binding, 0.2 micron filter. Infuse over ~4 hours; initiate at 1 mg/kg/hour. If tolerated, increase by 2 mg/kg/hour every 30 minutes to a maximum rate of 7 mg/kg/hour. For mild to moderate infusion reactions, decrease rate or temporarily hold; for severe hypersensitivity reactions or anaphylaxis, discontinue immediately and initiate appropriate medical treatment. Monitor vital signs prior to each rate increase.


Store intact vials between 2°C and 8°C (36°F and 46°F). Final solutions diluted in NS for infusion should be used immediately if possible but may be stored for up to 24 hours between 2°C and 8°C (36°F and 46°F); do not freeze. Protect from light.

Drug Interactions

There are no known significant interactions.

Adverse Reactions


Cardiovascular: Flushing (≤13%)

Dermatologic: Skin rash (infants and children: 18%; children, adolescents, and adults: ≥3%), urticaria (8% to 13%)

Hypersensitivity: Hypersensitivity reaction (including severe and immune-mediated)

Immunologic: Development of IgG antibodies (children, adolescents, and adults: 100%; infants and children: 89%; may affect efficacy)

Miscellaneous: Fever (infants and children: 15%; children, adolescents, and adults: ≥3%)

1% to 10%:

Cardiovascular: Chest discomfort (children, adolescents, and adults: 7%), hypertension (infants and children: 10%), increased blood pressure (children, adolescents, and adults: 5%), oxygen saturation decreased (infants and children: 8%), peripheral edema (children, adolescents, and adults: 3%), tachycardia (infants and children: 8%)

Dermatologic: Erythema of skin (infants and children: 5%), hyperhidrosis (children, adolescents, and adults: 8%), pallor (infants and children: 5%), papular rash (children, adolescents, and adults: 3%), pruritus (children, adolescents, and adults: 3%)

Gastrointestinal: Diarrhea (children, adolescents, and adults: ≥3%), nausea (≥3%), vomiting (5%)

Hypersensitivity: Anaphylaxis (children, adolescents, and adults: 7%)

Local: Local swelling (children, adolescents, and adults: ≥3%)

Nervous system: Agitation (infants and children: 5%), dizziness (≥3%), fatigue (≥3%), feeling hot (≤5%), headache (≥3%), paresthesia (children, adolescents, and adults: ≥3%), rigors (infants and children: 5%)

Neuromuscular & skeletal: Muscle twitching (children, adolescents, and adults: 7%), myalgia (children, adolescents, and adults: 5%), tremor (infants and children: 5%)

Respiratory: Cough (infants and children: 8%), cyanosis (infants and children: 5%), pharyngeal edema (children, adolescents, and adults: 3%), tachypnea (infants and children: 8%)

Frequency not defined:

Cardiovascular: Atrioventricular nodal arrhythmia, bradycardia, chest pain, hypotension, livedo reticularis, supraventricular tachycardia, ventricular premature contractions

Gastrointestinal: Retching

Hypersensitivity: Angioedema

Nervous system: Irritability

Neuromuscular & skeletal: Arthropathy

Ophthalmic: Increased lacrimation

Respiratory: Bronchospasm, respiratory distress


Cardiovascular: Cardiac failure, cold extremity

Dermatologic: Diaphoresis, skin necrosis (lesions; immune-mediated)

Endocrine & metabolic: Hyperparathyroidism, hypervolemia

Gastrointestinal: Abdominal pain

Genitourinary: Membranous glomerulonephritis

Hypersensitivity: Anaphylactic shock

Nervous system: Nervousness, seizure

Neuromuscular & skeletal: Back pain, muscle spasm

Respiratory: Apnea, dyspnea, hypoxia, rales, respiratory failure, stridor, wheezing

ALERT: U.S. Boxed Warning

Risk of hypersensitivity reactions:

Life-threatening anaphylactic reactions and severe hypersensitivity reactions, presenting as respiratory distress, hypoxia, apnea, dyspnea, bradycardia, tachycardia, bronchospasm, throat tightness, hypotension, angioedema (including tongue or lip swelling, periorbital edema, and face edema), and urticaria, have occurred in some patients during and after alglucosidase alfa infusions. Immune-mediated reactions presenting as proteinuria, nephrotic syndrome, and necrotizing skin lesions have occurred in some patients following alglucosidase alfa treatment. Closely observe patients during and after alglucosidase alfa administration and be prepared to manage anaphylaxis and hypersensitivity reactions. Inform patients of the signs and symptoms of anaphylaxis, hypersensitivity reactions, and immune-mediated reactions, and have them seek immediate medical care if signs and symptoms occur.

Risk of cardiorespiratory failure:

Infantile-onset Pompe disease patients with compromised cardiac or respiratory function may be at risk of serious acute exacerbation of cardiac or respiratory compromise due to fluid overload and require additional monitoring.


Concerns related to adverse effects:

• Hypersensitivity/Anaphylactoid reactions: [US Boxed Warning]: Life-threatening anaphylactic reactions and severe hypersensitivity reactions, some of which were IgE mediated, may occur; immediate medical support should be readily available. Reactions may occur during and up to 3 hours after infusion. Patients who develop IgE antibodies to alglucosidase alfa may be at a higher risk; monitor these patients closely during administration. Consider testing for IgG titers in patients who develop allergic reactions; may also test for IgE antibodies or other mediators of anaphylaxis. Consider risks/benefits of readministration following an anaphylactic or severe allergic reaction; some patients have been rechallenged under close clinical supervision; appropriate resuscitation measures should be available.

• IgG antibody formation: The presence of IgG antibodies has been observed within 3 months from the onset of therapy in the majority of patients. Patients with high and sustained IgG antibody titers, including cross-reactive immunologic material (CRIM)-negative patients, may result in reduced efficacy of alglucosidase alfa (eg, loss of motor function, ventilator dependence, death).

• Immune-mediated adverse effects: [US Boxed Warning]: Severe immune-mediated reactions (eg, necrotizing skin lesions, nephrotic syndrome secondary to membranous glomerulonephritis, proteinuria, inflammatory arthropathy) may occur; immediate medical support should be readily available. Reactions have occurred up to 3 years after initiation of therapy. Monitor urinalysis periodically. Monitor for immune-mediated reaction development. Consider testing for IgG titers in patients who develop immune-mediated reactions; may also test for IgE antibodies. Consider risks/benefits of readministration; some patients have been successfully rechallenged under close clinical supervision; immune tolerance induction administered in conjunction with alglucosidase alfa may aid tolerability of alglucosidase alfa in pediatric Pompe disease.

• Infusion reactions: Infusion-related reactions are common and may occur during and up to 2 hours after infusion. Appropriate medical support for the management of infusion reactions should be readily available. Discontinue immediately for severe hypersensitivity or anaphylactic reaction; mild to moderate reactions may be managed by reducing the infusion rate and/or administering antihistamines and/or antipyretics. Use caution with subsequent infusions; infusion reactions have occurred despite premedication with antihistamines, antipyretics, and/or corticosteroids. Patients with acute underlying illness are at greater risk for infusion reactions, including cardiorespiratory failure; monitor closely during infusion. Although less common, delayed-onset (within 48 hours after administration) infusion reactions have also occurred.

Disease-related concerns:

• Cardiovascular disease: [US Boxed Warning]: Use with caution in patients with compromised cardiac function; risk of acute cardiorespiratory failure secondary to infusion-related reactions or fluid overload may be increased. Additional monitoring is warranted. Cardiorespiratory failure has been observed in patients with cardiac hypertrophy up to 72 hours after infusion; ventricular arrhythmias and bradycardia have also been observed in patients with cardiac hypertrophy.

• Respiratory disease: [US Boxed Warning]: Use with caution in patients with compromised respiratory function; risk of acute cardiorespiratory failure secondary to infusion-related reactions or fluid overload may be increased. Additional monitoring is warranted.

• Sepsis: Patients with sepsis may be at increased risk for cardiorespiratory failure during infusions.

Special populations:

• Infantile-onset Pompe disease: Patients with infantile-onset Pompe disease should have a CRIM assessment early in their disease course and be managed by a clinical specialist knowledgeable in immune tolerance induction. Immune tolerance induction administered prior to and in conjunction with initiation of alglucosidase alfa has been reported to aid tolerability of alglucosidase alfa in CRIM-negative patients (CRIM-negative patients 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).

Dosage form specific issues:

• Polysorbate 80: Some dosage forms may contain polysorbate 80 (also known as Tweens). Hypersensitivity reactions, usually a delayed reaction, have been reported following exposure to pharmaceutical products containing polysorbate 80 in certain individuals (Isaksson 2002; Lucente 2000; Shelley 1995). Thrombocytopenia, ascites, pulmonary deterioration, and renal and hepatic failure have been reported in premature neonates after receiving parenteral products containing polysorbate 80 (Alade 1986; CDC 1984). See manufacturer's labeling.

Other warnings/precautions:

• Anesthesia: Use of general anesthesia for catheter placement for alglucosidase alfa infusions may be complicated by the presence of cardiac and skeletal (including respiratory) muscle weakness in patients with Pompe disease; use general anesthesia with caution. Ventricular arrhythmias and bradycardia, resulting in cardiac arrest or death or requiring cardiac resuscitation or defibrillation, have been observed in patients with cardiac hypertrophy.

• Registry: A registry has been created to monitor therapeutic responses and adverse effects during long-term treatment; patients should be encouraged to register (1-800-745-4447).

Monitoring Parameters

Liver enzymes (baseline and periodically; elevation may be due to disease process); vital signs (prior to each infusion rate increase) during and following infusion; immune-mediated reactions involving skin and other organs; volume overload; urinalysis (periodically)

The manufacturer recommends monitoring for IgG antibody formation every 3 months for 2 years, then annually. Consider testing if patient develops allergic or other suspected immune-mediated reaction or experiences loss of clinical response. Testing services for antibodies are available through Genzyme Corporation at 1-800-745-4447. Patients who experience anaphylactic or allergic reactions may also be tested for IgE antibodies to alglucosidase alfa and other mediators of anaphylaxis.

Reproductive Considerations

A registry has been established for Pompe patients; females of childbearing potential are encouraged to enroll in the registry.

Pregnancy Considerations

Use of alglucosidase alfa in females who continued enzyme replacement therapy throughout pregnancy is limited (de Vries 2011; Holbeck-Brendel 2017; Kłos 2017; Oliveira Santos 2018; Perniconi 2016; Van Houtte 2019; Zagnoli 2013). However, information related to Pompe disease in pregnancy is also limited (Karabul 2014).

Adverse events, such as worsening muscle weakness or decreased respiratory function, may be exacerbated by pregnancy and/or Pompe disease (Karabul 2014). The continuation of alglucosidase alfa during pregnancy may be considered (van der Ploeg 2017).

A registry has been established for Pompe patients; pregnant females are encouraged to enroll in the registry (1-800-745-4447).

Patient Education

What is this drug used for?

• It is used to treat Pompe disease.

All drugs may cause side effects. However, many people have no side effects or only have minor side effects. Call your doctor or get medical help if any of these side effects or any other side effects bother you or do not go away:

• Flu-like signs

• Diarrhea

• Constipation

• Trouble sleeping

WARNING/CAUTION: Even though it may be rare, some people may have very bad and sometimes deadly side effects when taking a drug. Tell your doctor or get medical help right away if you have any of the following signs or symptoms that may be related to a very bad side effect:

• Infusion reaction

• Kidney problems like unable to pass urine, blood in the urine, change in amount of urine passed, or weight gain

• Skin changes

• Shortness of breath

• Excessive weight gain

• Swelling of arm or leg

• Tremors

• Muscle pain

• Severe loss of strength and energy

• Signs of an allergic reaction, like rash; hives; itching; red, swollen, blistered, or peeling skin with or without fever; wheezing; tightness in the chest or throat; trouble breathing, swallowing, or talking; unusual hoarseness; or swelling of the mouth, face, lips, tongue, or throat.

Note: This is not a comprehensive list of all side effects. Talk to your doctor if you have questions.

Consumer Information Use and Disclaimer: This information should not be used to decide whether or not to take this medicine or any other medicine. Only the healthcare provider has the knowledge and training to decide which medicines are right for a specific patient. This information does not endorse any medicine as safe, effective, or approved for treating any patient or health condition. This is only a limited summary of general information about the medicine's uses from the patient education leaflet and is not intended to be comprehensive. This limited summary does NOT include all information available about the possible uses, directions, warnings, precautions, interactions, adverse effects, or risks that may apply to this medicine. This information is not intended to provide medical advice, diagnosis or treatment and does not replace information you receive from the healthcare provider. For a more detailed summary of information about the risks and benefits of using this medicine, please speak with your healthcare provider and review the entire patient education leaflet.

Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.