Aglucosidase (Lumizyme) for Treatment of Pompe disease (GAA Deficiency)

LUMIZYME (alglucosidase alfa) for injection, for intravenous use

Pompe disease (acid maltase deficiency, glycogen storage disease type II, GSD II, glycogenosis type II) is an inherited disorder of glycogen metabolism caused by the absence or marked deficiency of the lysosomal enzyme GAA. LUMIZYME provides an exogenous source of GAA. Binding to mannose-6-phosphate receptors on the cell surface has been shown to occur via carbohydrate groups on the GAA molecule, after which it is internalized and transported into lysosomes, where it undergoes proteolytic cleavage that results in increased enzymatic activity. It then exerts enzymatic activity in cleaving glycogen. 



Initial U.S. Approval: 2010


INDICATIONS AND USAGE
LUMIZYME (alglucosidase alfa) is a lysosomal glycogen-specific enzyme indicated for patients 8 years and older with late (non-infantile) onset Pompe disease (GAA deficiency) who do not have evidence of cardiac hypertrophy. The safety and efficacy of LUMIZYME have not been evaluated in controlled clinical trials in infantile-onset patients, or in late (non-infantile) onset patients less than 8 years of age

DOSAGE AND ADMINISTRATION
The recommended dosage of LUMIZYME is 20 mg/kg body weight administered every 2 weeks as an intravenous infusion.

DOSAGE FORMS AND STRENGTHS
Dosage form: Lyophilized powder for solution for intravenous infusion
Dosage strength: 5 mg/mL

CONTRAINDICATIONS
None

WARNINGS AND PRECAUTIONS
Life-threatening anaphylactic reactions have been observed in some patients during LUMIZYME infusions. Ensure that appropriate medical support measures, including cardiopulmonary resuscitation equipment are readily available when LUMIZYME is administered.
If severe allergic or anaphylactic reactions occur, consider immediate discontinuation of the administration of LUMIZYME and initiate appropriate medical treatment.
Severe cutaneous and systemic immune-mediated reactions can occur with alglucosidase alfa. Therefore, monitor patients for the development of systemic immune complex-mediated reactions involving skin and other organs while receiving LUMIZYME

ADVERSE REACTIONS
The most frequently reported adverse reactions (≥5%) in clinical studies were infusion reactions and included: anaphylaxis, urticaria, diarrhea, vomiting, dyspnea, pruritus, rash/erythema, pharyngolaryngeal pain, neck pain, hypoacusis, flushing/feeling hot, pain in extremity, fall and chest discomfort.

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