Brad, a Pompe Patient

Brad, a Pompe Patient


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

Treating Pompe Disease

Lumizyme® (alglucosidase alfa) is a medication that replaces a missing or deficient enzyme in people with Pompe disease.

If you have Pompe disease, ask your healthcare provider if Lumizyme is an appropriate treatment for you.

The information provided on this site is not intended to replace discussions with your healthcare provider. Be sure to talk to your healthcare provider if you have questions about Pompe disease, your Pompe treatment regimen, Lumizyme, or side effects you may have while taking Lumizyme.


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

Important Safety Information


LUMIZYME can cause serious side effects including:

Allergic Reactions Including Anaphylaxis and Immune-System Reactions:
Patients treated with LUMIZYME have experienced life-threatening allergic-type reactions, including anaphylaxis, and reactions related to the immune system, during and after infusion. Appropriate medical support equipment should be readily available when LUMIZYME is administered. Your healthcare provider may give you antihistamine and/or anti-fever medications before your infusions. You should seek immediate medical care if signs and symptoms of any severe reactions occur.

Signs of allergic reactions included cardiac and respiratory arrest (heart and lungs stop working), difficulty or shallow breathing; abnormal heart rate; low blood pressure; throat tightness; swelling of the tongue, lip, or face; and rash or hives.

Signs of an immune-system reaction included kidney dysfunction, including elevated protein levels in urine, and severe skin lesions (tissue death).

Depending on the severity of the reaction, your healthcare provider may stop or slow the infusion and provide appropriate medical care. You may require close observation during and after the LUMIZYME infusion.

Risk of Acute Cardiorespiratory Failure:
Patients with compromised heart or breathing function due to Pompe disease, may be at risk of sudden worsening of their heart or and/or lung function with LUMIZYME due to the fluid from the infusion. You may require close observation during and after your infusion.

Infusion Associated Reactions (IARs):Infusion Associated Reactions (IARs) have been observed in patients treated with Lumizyme. Your healthcare provider may decide to decrease the infusion rate, temporarily stop the infusion, or administer additional anti-fever and/or antihistamines medications that may treat the symptoms.

Risk of Irregular Heartbeat and Sudden Death during General Anesthesia for Central Venous Catheter Placement: Your healthcare provider should use caution when administering general anesthesia for the placement of a central venous catheter (a tubing line that can reach a large vein near your heart) for your LUMIZYME infusion.

Risk of Antibody Development: Patients with Infantile-onset Pompe disease (IOPD) should be managed by a specialist knowledgeable in immune tolerance induction (training the immune system to tolerate LUMIZYME) in Pompe disease to optimize treatment. Some patients who develop high and sustained IgG antibody levels may have a reduced response to LUMIZYME treatment.

Monitoring: Laboratory Tests:Your healthcare provider should monitor you for antibody formation every 3 months for 2 years, and then annually thereafter, depending on your response to treatment.

Adverse Reactions

The most frequently reported adverse reactions during LUMIZYME studies in patients were allergic reactions and included: anaphylaxis, rash, fever, flushing/feeling hot, hives, headache, excessive sweating, nausea, cough, less oxygen in the blood, fast heart rate, rapid breathing, chest discomfort, dizziness, muscle twitching, agitation, bluish or purple skin, redness of skin, high blood pressure/increased blood pressure, facial paleness, chills, tremor, vomiting, fatigue, and muscle pain.

Please see the Full Prescribing Information for complete details, including boxed WARNING.