Life-threatening anaphylactic reactions, severe allergic reactions and immune mediated reactions have been observed in some patients during Myozyme
infusions. Therefore, appropriate medical support should be readily available when Myozyme is administered.
Warnings and Precautions
Risk of Cardiorespiratory
Patients with compromised cardiac or
respiratory function may be at risk for serious acute
exacerbation of their cardiac or respiratory compromise due to
infusion reactions, and require additional monitoring.
MYOZYME (alglucosidase alfa), a lysosomal glycogen-specific enzyme, consists of the human enzyme acid alfa-glucosidase (GAA), encoded by the most predominant of nine observed haplotypes of this gene. MYOZYME is produced by recombinant DNA technology in a Chinese hamster ovary cell line.
MYOZYME® (alglucosidase alfa) [see Description] 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 an untreated historical control, whereas use of MYOZYME in patients with other forms of Pompe disease has not been adequately studied to assure safety and efficacy [see Clinical Studies ].
Published Studies Related to Myozyme (Alglucosidase Alfa)
A randomized study of alglucosidase alfa in late-onset Pompe's disease. [2010.04.15]
BACKGROUND: Pompe's disease is a metabolic myopathy caused by a deficiency of acid alpha glucosidase (GAA), an enzyme that degrades lysosomal glycogen. Late-onset Pompe's disease is characterized by progressive muscle weakness and loss of respiratory function, leading to early death. We conducted a randomized, placebo-controlled trial of alglucosidase alfa, a recombinant human GAA, for the treatment of late-onset Pompe's disease... CONCLUSIONS: In this study population, treatment with alglucosidase alfa was associated with improved walking distance and stabilization of pulmonary function over an 18-month period. (ClinicalTrials.gov number, NCT00158600.) Massachusetts Medical Society
Early treatment with alglucosidase alpha prolongs long-term survival of infants with Pompe disease. [2009.09]
In a previous 52-wk trial, treatment with alglucosidase alpha markedly improved cardiomyopathy, ventilatory function, and overall survival among 18 children <7 mo old with infantile-onset Pompe disease. Sixteen of the 18 patients enrolled in an extension study, where they continued to receive alglucosidase alpha at either 20 mg/kg biweekly (n = 8) or 40 mg/kg biweekly (n = 8), for up to a total of 3 y...
High antibody titer in an adult with Pompe disease affects treatment with alglucosidase alfa. [2010.12]
Clinical trials have demonstrated beneficial effects of enzyme replacement therapy (ERT) with alglucosidase alfa in infants, children and adults with Pompe disease. Recent studies have shown that high antibody titers can occur in patients receiving ERT and counteract the effect of treatment... This case demonstrates that also patients with an appreciable amount of properly folded and catalytically active endogenous acid alpha-glucosidase can develop antibodies against alglucosidase alfa that affect the response to ERT.
Enzyme replacement therapy with alglucosidase alfa in 44 patients with late-onset glycogen storage disease type 2: 12-month results of an observational clinical trial. [2010.01]
Late-onset glycogen storage disease type 2 (GSD2)/Pompe disease is a progressive multi-system disease evoked by a deficiency of lysosomal acid alpha-glucosidase (GAA) activity. GSD2 is characterized by respiratory and skeletal muscle weakness and atrophy, resulting in functional disability and reduced life span...
A randomized study of alglucosidase alfa in late-onset Pompe's disease. 
treatment of late-onset Pompe's disease... CONCLUSIONS: In this study population, treatment with alglucosidase alfa was
Clinical Trials Related to Myozyme (Alglucosidase Alfa)
A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Patients With Infantile-Onset Pompe Disease Who Have Never Been Treated [Recruiting]
A study to demonstrate comparable safety, efficacy, and pharmacokinetics (PK) of
alglucosidase alfa manufactured at the 160 L and 4000 L scales in patients who have been
diagnosed with infantile-onset Pompe disease. Patients will be treated with alglucosidase
alfa 160 L scale product in the US and 4000 L scale product in the regions outside the US.
An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive CRIM (-) Patients With Infantile-Onset Pompe Disease [Recruiting]
An exploratory, open-labeled study of patients with Infantile-Onset Pompe disease to
evaluate the efficacy, clinical benefit and safety of prophylactic immunomodulatory regimen
of Rituximab and Methotrexate prior to Myozyme infusion.
A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease [Recruiting]
The objective of this study is to evaluate the efficacy and safety of treatment with 4000 L
alglucosidase alfa (Lumizyme®) in Pompe patients.
Alglucosidase Alfa Temporary Access Program [Available]
Pompe disease (also known as glycogen storage disease Type II) is caused by a deficiency of
a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by
the body's cells to break down glycogen (a stored form of sugar) within specialized
structures called lysosomes. In patients with Pompe disease, an excessive amount of
glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle,
which prevents their normal function. The objective of this expanded access study is to
provide patients with Pompe disease in the United States (US), access to alglucosidase alfa
produced from a scaled up manufacturing process for a limited time until production at this
scale is approved for commercial use by the Food and Drug Administration.
Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa [Recruiting]
This is an open-label, multicenter study of patients with late-onset Pompe disease naïve to
treatment with enzyme replacement therapy (ERT). The primary purpose of this study is to
evaluate glycogen clearance in muscle tissue samples collected pre and post alglucosidase
alfa treatment in patients with Late-Onset Pompe disease.
Reports of Suspected Myozyme (Alglucosidase Alfa) Side Effects
Respiratory Failure (15),
Disease Progression (14),
Cardiac Failure (12),
Hypertrophic Cardiomyopathy (11),
Respiratory Disorder (9),
Drug Ineffective (9),
Cardio-Respiratory Arrest (8),
Pneumonia (8), more >>
Page last updated: 2013-02-10