Inborn errors of metabolism:

Indications for LUMIZYME:

Pompe disease (GAA deficiency).

Adults and Children:

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.

Boxed Warning:

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

Warnings/Precautions:

Risk of anaphylaxis or severe hypersensitivity 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 or compromised cardiac and/or respiratory function: 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. Labor & delivery. Pregnancy. Nursing mothers (consider interrupting breastfeeding, pumping and discarding breast milk during and for 24hrs after therapy).

Pharmacologic Class:

Hydrolytic lysosomal glycogen-specific enzyme.

Adverse Reactions:

Hypersensitivity reactions (including anaphylaxis, rash, pyrexia, flushing/feeling hot, urticaria, headache, hyperhidrosis, nausea, cough, decreased oxygen saturation, tachycardia, tachypnea, chest discomfort, dizziness, muscle twitching, agitation, cyanosis, erythema, hypertension, pallor, rigors, tremor, vomiting, fatigue, myalgia).

Note:

To enroll in the Pompe registry call (800) 745-4447.

Generic Availability:

NO

How Supplied:

Single-use vial—1, 10