Lumizyme® (alglucosidase alfa) is a medication that replaces the enzyme acid alfa glucosidase (GAA), which is thought to be missing or deficient in people with Pompe disease. Since people with Pompe disease do not produce enough of the GAA enzyme on their own, a replacement enzyme may be needed.
Lumizyme is an enzyme replacement therapy (ERT) produced using recombinant DNA technology, a process that enables animal cells to produce human enzymes. The genetically engineered enzyme is intended to act like the naturally occurring GAA that is normally produced within human cells. Treatment with Lumizyme is not a cure for Pompe disease; that is, it may provide replacement for the missing enzyme, but it will not correct the underlying genetic defect that causes the enzyme deficiency.
Life-threatening severe allergic (anaphylactic) and hypersensitivity reactions have occurred in some patients during Lumizyme infusions and up to 3 hours after Lumizyme infusion. Your doctor should have appropriate medical support measures, including cardiopulmonary (heart/lung) resuscitation equipment readily available during your Lumizyme infusion.
If you experience severe allergic reactions, your doctor may stop the infusion and will provide appropriate medical care.
Severe skin-related (cutaneous) and other immune system-related reactions can occur within several weeks to 3 years after starting treatment with Lumizyme. Your doctor should monitor you for the development of reactions involving skin and other organs.
Download this document for more details on Lumizyme, its indication, safety information, and its usage:
LUMIZYME® (alglucosidase alfa) is an enzyme replacement therapy for patients with Pompe disease (acid α-glucosidase (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 such a reaction is severe enough, your doctor may decide to immediately discontinue the infusion and provide you with immediate medical care. Appropriate medical support and monitoring measures should be available during infusion.
Immune-Mediated Reactions: You or your child may be monitored for the development of systemic immune-mediated reactions while receiving Lumizyme. If these reactions occur, your doctor may discontinue the infusion and initiate appropriate medical treatment.
Risk of Acute Cardiorespiratory Failure: Infant Pompe patients with heart or breathing problems who are experiencing an acute respiratory condition may be at risk for increasing the seriousness of these problems as a result of Lumizyme administration due to the infusion fluid, and your child’s doctor may require additional monitoring for these infants.
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 Lumizyme infusion. Ventricular arrhythmias and slow heart rate resulting in cardiac arrest or death have been observed in infant Pompe patients with cardiac hypertrophy during general anesthesia for central venous catheter placement. Appropriate medical support and monitoring measures should be available during infusion.
Risk of Antibody Development: Patients with infantile-onset Pompe disease (IOPD) should be managed by a clinical specialist knowledgeable in immune tolerance induction in Pompe disease to optimize treatment. Some patients who develop high and sustained IgG antibody levels, including certain IOPD patients, may have a reduced response to Lumizyme.
Monitoring: Laboratory Tests: Patients should be monitored for IgG antibody formation every 3 months for 2 years and then annually thereafter.
The most frequently reported adverse reactions during Lumizyme studies in patients were allergy 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.