INDICATIONS AND USAGE

MYOZYME (alglucosidase alfa) is a lysosomal glycogen-specific enzyme indicated for use in patients with Pompe disease (GAA deficiency).MYOZYME has been shown to improve ventilator-free survival in patients with infantile-onset Pompe disease as compared to an untreated historical control, whereas use of MYOZYME in patients with other forms of Pompe disease has not been adequately studied to assure safety and efficacy. (1)

DOSAGE AND ADMINISTRATION

• The recommended dosage of MYOZYME is 20 mg/kg body weight administered every 2 weeks as an intravenous infusion. (2)

DOSAGE FORMS AND STRENGTHS

• Dosage form: Lyophilized powder for solution for intravenous infusion.
• Dosage strength: 5 mg/mL. (3)

CONTRAINDICATIONS

• None. (4)

WARNINGS AND PRECAUTIONS

• Life-threatening anaphylactic reactions, severe allergic reactions and immune mediated reactions have been observed in some patients during and within three hours after MYOZYME infusions. Ensure that appropriate medical support measures, including cardiopulmonary resuscitation equipment, are readily available when MYOZYME is administered. If severe allergic or anaphylactic reactions occur, consider immediate discontinuation of the administration of MYOZYME and initiate appropriate medical treatment. (5.1)
• Patients with compromised cardiac or respiratory function require additional monitoring during MYOZYME infusions. Acute cardiorespiratory failure requiring intubation and inotropic support has been observed up to 72 hours after infusion. (5.2)
• Cardiac arrhythmias resulting in cardiac arrest or death, or requiring cardiac resuscitation or defibrillation have been observed in infantile-onset Pompe disease patients with cardiac hypertrophy during general anesthesia for central venous catheter placement. Caution should be used when administering general anesthesia in infantile-onset Pompe disease patients with cardiac hypertrophy.
• Severe cutaneous and systemic immune mediated reactions can occur with MYOZYME. Therefore, monitor patients for the development of systemic immune complex-mediated reactions involving skin and other organs while receiving MYOZYME. (5.5)

ADVERSE REACTIONS

• The most common serious treatment-emergent adverse reactions (occurring in > 10% of patients) observed in clinical studies with MYOZYME were pneumonia, respiratory failure, respiratory distress, catheter-related infection, respiratory syncytial virus infection, gastroenteritis and fever. (6.1)
• The most common reactions requiring intervention were infusion reactions. (6.1)

To report SUSPECTED ADVERSE REACTIONS, contact Genzyme at 1-800-745-4447 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch

USE IN SPECIFIC POPULATIONS

• Pregnancy: Physicians are encouraged to enroll pregnant patients in the Pompe Registry. (8.1, 17)
• Nursing Mothers: Physicians are encouraged to enroll nursing patients in the Pompe Registry. (8.3, 17)