Aglucosidase (Myozyme)for the Treatment of Pompe Disease

                  Aglucosidase (Myozyme)for the Treatment of Pompe Disease

MYOZYME® (alpha-l-glucosidase alfa) Injectable for intravenous infusion:

Pompe disease (glycogen storage disease type II, GSD II, glycogenosis type II, acid maltase deficiency) is an inherited disorder of glycogen metabolism caused by the absence or marked deficiency of the lysosomal enzyme GAA. 
MYOZYME 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: 2006

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 untreated historical control, whereas the use of MYOZYME in patients with other forms of Pompe disease has not been adequately studied to assure safety and efficacy.

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

Dosage form: Lyophilized powder for solution for intravenous infusion. 
Do sage st ren gt h: 5 mg/mL. 


Life-threatening anaphylactic, severe allergic and immune mediated reactions: Ensure that appropriate medical support is readily available. If severe allergic or anaphylactic reactions occur, consider immediate discontinuation of MYOZYME and initiate appropriate medical treatment.
Severe cutaneous and systemic immune-mediated reactions: Monitor patients for the development of systemic immune-mediated reactions involving skin and other organs. 
Acute cardiorespiratory failure: Patients with compromised cardiac or respiratory function may be at risk of acute cardiorespiratory failure. Appropriate medical support and monitoring measures should be readily available.
Cardiac arrhythmias and sudden cardiac death during general anesthesia: Caution should be used when administering general anesthesia for the placement of a central venous catheter intended for MYOZYME infusion. 

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. 
The most common reactions requiring intervention were infusion reactions.