Version July 2025
Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Initiate alglucosidase alfa in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (eg, anaphylaxis) occurs, discontinue alglucosidase alfa and immediately initiate appropriate medical treatment, including use of epinephrine. Inform patients of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur.
Immune-mediated reactions presenting as proteinuria, nephrotic syndrome, and necrotizing skin lesions have occurred in some patients following alglucosidase alfa treatment. Monitor patients for the development of systemic immune-mediated reactions involving skin and other organs while receiving alglucosidase alfa.
Infantile-onset Pompe disease (IOPD) 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 and require additional monitoring.
Dosage guidance:
Clinical considerations: Consider antihistamines, antipyretics, and/or corticosteroids premedications prior to administering. Have appropriate medical monitoring and support measures, including CPR equipment, readily available during administration.
Pompe disease: Noninfantile, late onset: IV: 20 mg/kg every 2 weeks.
Missed dose: If a dose is missed, administer as soon as possible and continue the regular dosing schedule (with at least 2 weeks between doses).
There are no dosage adjustments provided in the manufacturer's labeling.
There are no dosage adjustments provided in the manufacturer's labeling.
(For additional information see "Alglucosidase alfa: Pediatric drug information")
Dosage guidance:
Safety: Pretreatment with antihistamines, antipyretics, and/or corticosteroids can be considered to reduce risk of infusion reactions. Initiate in a health care setting that includes cardiopulmonary monitoring and can provide resuscitation if necessary.
Clinical considerations: 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.
Pompe disease, infantile onset: Infants, Children, and Adolescents: IV: 20 mg/kg/dose every 2 weeks. Note: For patients being rechallenged after a severe hypersensitivity reaction, consider a dose reduction.
Pompe disease, late onset; noninfantile: Children and Adolescents: IV: 20 mg/kg/dose every 2 weeks. Note: For patients being rechallenged after a severe hypersensitivity reaction, consider a dose reduction.
There are no dosage adjustments provided in the manufacturer's labeling.
There are no dosage adjustments provided in the manufacturer's labeling.
The following adverse drug reactions and incidences are derived from product labeling unless otherwise specified. Adverse reactions reported in infants, children, adolescents, or adults.
>10%:
Cardiovascular: Flushing
Dermatologic: Skin rash, urticaria
Hypersensitivity: Hypersensitivity reaction (including anaphylaxis, angioedema, severe hypersensitivity reaction, type III hypersensitivity reaction)
Immunologic: Antibody development (including development of IgG antibodies)
Miscellaneous: Fever
1% to 10%:
Cardiovascular: Chest discomfort, hypertension, peripheral edema, tachycardia
Dermatologic: Erythema of skin, hyperhidrosis, pallor, papular rash, pruritus
Gastrointestinal: Nausea, vomiting
Nervous system: Agitation, fatigue, feeling hot, rigors, tremor
Neuromuscular & skeletal: Muscle twitching, myalgia
Respiratory: Cough, cyanosis, oxygen saturation decreased, pharyngeal edema, tachypnea
Postmarketing:
Cardiovascular: Acute cardiorespiratory failure
Dermatologic: Skin necrosis (lesions)
Endocrine & metabolic: Hyperparathyroidism, hypervolemia
Genitourinary: Proteinuria
Hypersensitivity: Anaphylactic shock, infusion-related reaction
Renal: Membranous glomerulonephritis, nephrotic syndrome
There are no contraindications listed in the US manufacturer's labeling.
Canadian labeling: Hypersensitivity to alglucosidase alfa or any component of the formulation.
Concerns related to adverse effects:
• Hypersensitivity/Anaphylactoid reactions: 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: Reactions have occurred up to 3 years after initiation of therapy. 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, including arthralgia, chills, dyspnea, erythema, fatigue, fever, flu-like symptoms, flushing, headache, myalgia, nausea, pain, rash, syncope, tachycardia, and urticaria, may occur. Appropriate medical support for the management of infusion reactions should be readily available. Use caution with subsequent infusions; infusion reactions have occurred despite premedication with antihistamines, antipyretics, and/or corticosteroids.
Disease-related concerns:
• Cardiovascular disease: Cardiorespiratory failure has been observed in patients with cardiac hypertrophy; ventricular arrhythmias and bradycardia have also been observed in patients with cardiac hypertrophy.
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). A limited number of CRIM-positive patients have had high and sustained IgG antibody titers, usually with very low endogenous enzyme; manage these patients with a specialist knowledgeable in immune tolerance induction in Pompe disease.
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).
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]
No
Solution (reconstituted) (Lumizyme Intravenous)
50 mg (per each): $1,192.03
Disclaimer: A representative AWP (Average Wholesale Price) price or price range is provided as reference price only. A range is provided when more than one manufacturer's AWP price is available and uses the low and high price reported by the manufacturers to determine the range. The pricing data should be used for benchmarking purposes only, and as such should not be used alone to set or adjudicate any prices for reimbursement or purchasing functions or considered to be an exact price for a single product and/or manufacturer. Medi-Span expressly disclaims all warranties of any kind or nature, whether express or implied, and assumes no liability with respect to accuracy of price or price range data published in its solutions. In no event shall Medi-Span be liable for special, indirect, incidental, or consequential damages arising from use of price or price range data. Pricing data is updated monthly.
Excipient information presented when available (limited, particularly for generics); consult specific product labeling.
Solution Reconstituted, Intravenous:
Myozyme: 50 mg (1 ea) [contains polysorbate 80]
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.
Note: Consider premedication with antihistamines, antipyretics, and/or corticosteroids prior to start of infusion. Initiate infusion in a health care setting that includes cardiopulmonary monitoring and can provide resuscitation if necessary.
Parenteral: IV infusion: Administer as a weight-based IV infusion over ~4 hours: initiate at ≤1 mg/kg/hour, then increase by 2 mg/kg/hour every 30 minutes as tolerated to a maximum rate of 7 mg/kg/hour. Infuse through a low protein-binding, 0.2 micron in-line filter. Do not administer products with visualized particulate matter. Protect from light.
Rate adjustment for infusion-related reactions or hypersensitivity:
Hypersensitivity:
Mild to moderate hypersensitivity: Slowing of infusion rate or temporary interruption of the infusion has been shown to treat symptoms. If infusion is temporarily stopped, it should be stopped for at least 30 minutes to allow for resolution of symptoms. If symptoms resolve, infusion may be resumed at half the previous rate at which symptoms occurred or lower, for 30 minutes. If tolerated, the infusion rate may be gradually increased. Monitor closely for recurrence of symptoms (Ref).
Anaphylaxis or severe hypersensitivity: Discontinue therapy immediately and initiate appropriate medical treatment (eg, antihistamines, corticosteroids, IV fluids, oxygen, or epinephrine). Patients may be rechallenged with extreme caution. For subsequent infusions, slower infusion rates in addition to a reduced dose are suggested (Ref).
Infusion-related reaction:
Mild to moderate infusion-related reaction: Slowing of infusion rate or temporary interruption of the infusion has been shown to treat symptoms; additional doses of antihistamines and antipyretics may be necessary. If infusion is temporarily stopped, it should be stopped for at least 30 minutes to allow for resolution of symptoms. If symptoms resolve, infusion may be resumed at half the previous rate at which symptoms occurred or lower, for 30 minutes. If tolerated, the infusion rate may be gradually increased. Monitor closely for recurrence of symptoms (Ref).
Severe infusion-related reaction: Discontinue therapy immediately and initiate appropriate medical treatment (eg, antihistamines, corticosteroids, IV fluids, oxygen, or epinephrine). Patients may be rechallenged with extreme caution (Ref).
Pompe disease: For use in patients with Pompe disease (acid alpha-glucosidase [GAA] deficiency).
Alglucosidase alfa may be confused with agalsidase alfa, agalsidase beta, alglucerase
None known.
There are no known significant interactions.
A registry has been established for patients diagnosed with Pompe disease; patients who may become pregnant are encouraged to enroll in the registry.
Use of alglucosidase alfa in patients who continued enzyme replacement therapy throughout pregnancy is limited (de Vries 2011; Goker-Alpan 2020; Holbeck-Brendel 2017; Kłos 2017; Koyuncu 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 patients diagnosed with Pompe disease; pregnant patients and patients of reproductive potential are encouraged to enroll in the registry (1-800-745-4447, extension 15500 or http://www.registrynxt.com).
Acid alfa-glucosidase is endogenous to breast milk; concentrations are lower in women with Pompe disease.
Following an infusion of alglucosidase alfa in one woman with Pompe disease, maximum enzyme activity was found in breast milk 2.5 hours after the dose and was ~0.3% of the maternal peak plasma value. Activity in breast milk returned to baseline values within 24 hours after the infusion (de Vries 2011).
According to the manufacturer, the decision to breastfeed during therapy should consider the risk of infant exposure, benefits of breastfeeding to the infant, and benefits of treatment to the mother; exposure may be minimized by temporarily expressing and discarding breast milk for 24 hours after administration.
A registry has been established for patients with Pompe disease; patients who are breastfeeding are encouraged to enroll in the registry (1-800-745-4447, extension 15500)
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 strongly recommends a baseline serum anti-alglucosidase alfa antibody (antidrug antibody [ADA]) prior to the first infusion. In patients with infantile-onset Pompe disease, monitor for ADA every 3 months for the first year. In patients with late-onset Pompe disease, monitor for ADA within 6 months of initiation and then as clinically needed. Consider testing for IgG ADA, IgE ADA, and other mediators if patient develops hypersensitivity reactions. Consider testing for IgG ADA in patients that develop other immune-mediated reactions. Consider testing for IgG ADA and inhibitory antibody activity in patients who experience loss of or reduced 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.
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.
Distribution: 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
Clearance: Higher mean clearance (50%) was observed in patients who tested positive for antibodies to alglucosidase alfa.
