There are numerous resources available to both healthcare providers and their patients to help contribute to their understanding of Pompe disease. Professional and patient organizations can help educate and bring together those in the Pompe community, and Sanofi Genzyme offers years of expertise and resources on treating, managing, and living with the disease. Sanofi Genzyme is committed to providing practitioners with information and access to resources that continue to help improve standards of care for patients with Pompe disease.
The Pompe Registry is an ongoing, observational database sponsored by Sanofi Genzyme that tracks the natural history and outcomes of patients with Pompe disease. All patients with Pompe disease are eligible for enrollment irrespective of their treatment status, and all physicians managing patients with Pompe disease are encouraged to learn about the Pompe Registry.
To learn more about the Pompe Registry
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.
Risk of Acute Cardiorespiratory Failure: Patients with acute underlying respiratory illness and compromised cardiac and/or respiratory function may be at risk of acute cardiorespiratory failure. Caution should be exercised when administering alglucosidase alfa to patients susceptible to fluid volume overload. Appropriate medical support and monitoring measures should be available during infusion and some patients may require longer observation times.
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 alglucosidase alfa infusion.
Risk of Antibody Development: As with all therapeutic proteins, there is potential for immunogenicity. There is some evidence to suggest that some patients who develop high and sustained IgG antibody titers may experience reduced clinical efficacy. 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.
Pregnancy: Based on animal data, alglucosidase alfa may cause fetal harm.
Please see the Full Prescribing Information for complete details, including boxed WARNING.