Treating Patients

The recommended dosage of Lumizyme is 20 mg/kg body weight administered every 2 weeks.

Indication

Lumizyme^® (alglucosidase alfa) is a hydrolytic lysosomal glycogen-specific enzyme indicated for patients with Pompe disease (GAA deficiency).

Treating Patients

The recommended dosage of Lumizyme is 20 mg/kg body weight administered every 2 weeks

Administered by Intravenous Infusion Lumizyme should be reconstituted, diluted and administered by a healthcare professional. Before administering to patients, Lumizyme must be

Reconstituted with sterile water for injection
Diluted with 0.9% sodium chloride for injection

About Pompe Disease

Pompe disease—also known as acid maltase deficiency or glycogen storage disease type II—is a progressive, debilitating, and often fatal neuromuscular disorder. It was first identified in 1932 by Johannes C. Pompe.³

Caused by a deficiency of the enzyme acid alfa glucosidase (GAA), Pompe disease is characterized by progressive glycogen accumulation primarily in the lysosomes of muscle cells. It is an autosomal recessive genetic disorder, affecting both sexes equally.

Early Diagnosis Is Key

Pompe disease is progressive, even in the absence of overt signs and symptoms
Early diagnosis is important to disease management
Pompe disease symptoms can be seen in other neuromuscular disorders, so a definitive diagnosis is important to determine appropriate treatment options
Fortunately, a diagnosis can be confirmed by enzyme assay demonstrating low GAA activity and by GAA gene sequencing

Indication

LUMIZYME^® (alglucosidase alfa) is a hydrolytic lysosomal glycogen-specific enzyme indicated for patients with Pompe disease (GAA deficiency).

Important Safety Information

WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS, IMMUNE-MEDIATED REACTIONS, AND RISK OF ACUTE CARDIORESPIRATORY FAILURE

Hypersensitivity Reactions Including Anaphylaxis
Patients treated with LUMIZYME have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Appropriate medical monitoring and support measures, including cardiopulmonary resuscitation equipment, should be readily available during LUMIZYME administration. If a severe hypersensitivity reaction (e.g. anaphylaxis) occurs, discontinue LUMIZYME immediately and initiate appropriate medical treatment.

Consider risks and benefits of re-administering LUMIZYME following severe hypersensitivity reactions. If a mild or moderate hypersensitivity reaction occurs, the infusion rate may be slowed or temporarily stopped. Prior to LUMIZYME administration, consider pretreating with antihistamines, antipyretics, and/or corticosteroids.

Immune-Mediated Reactions
Immune-mediated reactions presenting as proteinuria, nephrotic syndrome, and necrotizing skin lesions have occurred in some patients following LUMIZYME treatment. Monitor patients for the development of systemic immune-mediated reactions involving skin and other organs while receiving LUMIZYME.

Risk of Acute Cardiorespiratory Failure
Infantile-onset Pompe disease patients with compromised cardiac or respiratory function may be at risk of serious acute exacerbation of their cardiac or respiratory compromise due to fluid overload and require additional monitoring.

Infusion Associated Reactions (IARs): Infusion Associated Reactions (IARs) have been observed in patients treated with Lumizyme. Discontinue immediately or adjust the infusion rate and provide medical treatment based on the severity of the reaction. Closely monitor patients who have experienced IARs when re-administering LUMIZYME.

Risk of Cardiac Arrhythmia and Sudden Cardiac Death during General Anesthesia for Central Venous Catheter Placement: Caution should be used when administering general anesthesia for the placement of a central venous catheter intended for LUMIZYME infusion.

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 specialist knowledgeable in immune tolerance induction in Pompe disease to optimize treatment. Evidence suggests that patients who develop high and sustained IgG antibody titers may experience reduced clinical efficacy.

Monitoring: Laboratory Tests: Patients should be monitored for IgG antibody formation every 3 months for 2 years and then annually thereafter. Patients who experience hypersensitivity reactions, including anaphylaxis, may also be tested for IgE antibodies to LUMIZYME and other mediators of anaphylaxis.

Adverse Reactions

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.