By providing a replacement for the missing or deficient GAA enzyme, Lumizyme® (alglucosidase alfa) can be an important part of a care plan for patients with Pompe disease (GAA deficiency). Lumizyme is administered by intravenous infusion, and should be reconstituted, diluted, and administered by a health care professional.
Lumizyme is a hydrolytic lysosomal glycogen-specific enzyme indicated for patients with Pompe disease (acid α‑glucosidase (GAA) deficiency).
Because of the potential risks of infusion reactions as well as more serious adverse events, close monitoring is necessary for patients receiving Lumizyme treatment. Life-threatening anaphylactic and severe allergic reactions have been observed in some patients during and up to three hours after Lumizyme infusions. Appropriate medical support should always be readily available whenever Lumizyme is administered. Patients should be observed during and after each infusion by appropriate medical personnel familiar with Pompe disease and potential reactions to treatment.1
The recommended dosage of Lumizyme is 20 mg per kg of body weight, administered every two weeks as an intravenous infusion. Lumizyme should be reconstituted, diluted and administered by a healthcare professional. Infusions should be administered in a step-wise manner using an infusion pump, with patients carefully monitored throughout the approximately 4 hour process.
See full Prescribing Information for complete Instructions for Use and Reconstitution, Dilution, and Administration information.
LUMIZYME® (alglucosidase alfa) is a hydrolytic lysosomal glycogen-specific enzyme indicated for patients with Pompe disease (GAA deficiency).
WARNING: RISK OF ANAPHYLAXIS, HYPERSENSITIVITY AND IMMUNE-MEDIATED REACTIONS, AND RISK OF CARDIORESPIRATORY FAILURE
Anaphylaxis and Hypersensitivity Reactions: Life-threatening anaphylaxis and hypersensitivity reactions have been observed in some patients during and after treatment with alglucosidase alfa. If anaphylaxis or severe hypersensitivity reactions occur, immediately discontinue infusion and institute appropriate medical treatment. Appropriate medical support and monitoring measures should be available during infusion.
Immune-Mediated Reactions: Monitor patients for the development of systemic immune-mediated reactions involving skin and other organs. If immune-mediated reactions occur, consider discontinuation of the administration of alglucosidase alfa, and initiate appropriate medical treatment.
Risk of Acute Cardiorespiratory Failure: Patients with acute underlying respiratory illness or compromised cardiac and/or respiratory function may be at risk of serious exacerbation of their cardiac or respiratory compromise during infusions. Appropriate medical support and monitoring measures should be readily available during alglucosidase alfa infusion, and some patients may require prolonged observation times that should be individualized based on the needs of the patient.
Risk of Cardiac Arrhythmia and Sudden Cardiac Death during General Anesthesia for Central Venous Catheter Placement: Administration of general anesthesia can be complicated by the presence of severe cardiac and skeletal (including respiratory) muscle weakness. Therefore, caution should be used when administering general anesthesia. Ventricular arrhythmias and bradycardia, 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.
Risk of Antibody Development: Patients with infantile-onset Pompe disease should have a cross-reactive immunologic material (CRIM) assessment early in their disease course and be managed by a clinical specialist knowledgeable in immune tolerance induction in Pompe disease to optimize treatment. CRIM status has been shown to be associated with immunogenicity and patients’ responses to enzyme replacement therapies. There is evidence to suggest that some patients who develop high and sustained IgG antibody titers, including CRIM-negative patients, may experience reduced clinical alglucosidase alfa treatment efficacy.
Monitoring: Laboratory Tests: Patients should be monitored for IgG antibody formation every 3 months for 2 years and then annually thereafter.
The most frequently reported adverse reactions (≥ 5%) in clinical trials were hypersensitivity reactions and included: 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/increased blood pressure, pallor, rigors, tremor, vomiting, fatigue, and myalgia.
Please see the Full Prescribing Information for complete details, including boxed WARNING.