Inborn errors of metabolism:

Indications for MYOZYME:

Pompe disease (GAA deficiency).


Not applicable.


Give by IV infusion over 4 hours. Initial infusion rate: 1mg/kg/hr, may increase to 2mg/kg/hr every 30 minutes after tolerance established to max 7mg/kg/hr. 20mg/kg every 2 weeks.

Boxed Warning:

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


Discontinue immediately if severe anaphylactic or allergic reactions develop; have appropriate medical support measures available and during rechallenge. Increased risk of acute cardiorespiratory failure with acute underlying respiratory illness, compromised cardiac function, or sepsis. 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. Monitor for development of systemic immune complex-mediated reactions; consider discontinuing if occurs. Monitor for IgG antibody formation every 3 months for 2 years, then annually. Perform LFTs prior to initiation; monitor periodically. Labor & delivery. Pregnancy (Cat.B). Nursing mothers.

Pharmacologic Class:

Recombinant human alpha-glucosidase enzyme.

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).

How Supplied:

Single-use vial (50mg)—1