• Inborn errors of metabolism

Myozyme Generic Name & Formulations

General Description

Alglucosidase alfa 50mg/vial; lyophilized pwd for IV infusion after reconstitution and dilution; preservative-free.

Pharmacological Class

Recombinant human alpha-glucosidase enzyme.

How Supplied

Single-use vial—1


Myozyme Indications


Pompe disease (GAA deficiency).

Myozyme Dosage and Administration


Not applicable.


Give by IV infusion over 4hrs. 20mg/kg every 2 weeks. Initial infusion rate: max 1mg/kg/hr; may increase by 2mg/kg/hr every 30mins, if tolerated, to max 7mg/kg/hr.

Myozyme Contraindications

Not Applicable

Myozyme Boxed Warnings

Boxed Warning

Risk of anaphylaxis, severe allergic, and immune-mediated reactions. Risk of cardiorespiratory failure.

Myozyme Warnings/Precautions


Risk of anaphylaxis or severe allergic reactions; discontinue immediately if develop. Have appropriate medical support available and during rechallenge. Monitor for systemic immune-mediated reactions; consider discontinuing and treat if occur. Obtain urinalysis periodically. Acute underlying respiratory illness, compromised cardiac function, sepsis: risk of acute cardiorespiratory failure. Cardiac hypertrophy (increased risk of cardiac arrhythmia when given general anesthesia for central venous catheter placement). Infants with cardiac dysfunction may require prolonged observation times. Risk of antibody development. Infantile-onset disease: perform cross-reactive immunologic material (CRIM) assessment early in the disease course. Monitor for IgG antibody formation every 3 months for 2 years, then annually. Perform LFTs prior to initiation; monitor periodically. Pregnancy. Nursing mothers (consider interrupting breastfeeding, pumping and discarding breast milk during and for 24hrs after therapy).

Myozyme Pharmacokinetics

See Literature

Myozyme Interactions

Not Applicable

Myozyme Adverse Reactions

Adverse Reactions

Pneumonia, respiratory failure/distress, catheter-related infection, respiratory syncytial virus infection, gastroenteritis, fever; infusion reactions (temporarily stop or reduce infusion rate; discontinue if severe).

Myozyme Clinical Trials

See Literature

Myozyme Note

Not Applicable

Myozyme Patient Counseling

See Literature