Lumizyme® (alglucosidase alfa) is medication that replaces the enzyme acid alfa glucosidase (GAA), which is 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 regular supply of the replacement enzyme may help manage the disease in some patients.
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 a long-term, ongoing 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.
LUMIZYME® (alglucosidase alfa) is an enzyme replacement therapy for patients with Pompe disease (acid α-glucosidase (GAA) deficiency).
IMPORTANT SAFETY INFORMATION
If you or your child is taking Lumizyme, you should know that severe and potentially life-threatening allergic-type reactions known as anaphylaxis or severe hypersensitivity reactions, and other severe reactions related to the immune system may occur during and after Lumizyme treatment. Reactions may include, for example, kidney dysfunction, extreme difficulty breathing, shallow breathing, abnormal heart rate, low blood pressure, or throat tightness (including face, tongue and lip swelling) and skin lesions.
If you or your child has previously experienced these severe reactions, from any cause, you may require close observation during and after Lumizyme administration. You should discuss with your physician the signs and symptoms of anaphylaxis and hypersensitivity reactions. Anaphylaxis or severe hypersensitivity reactions are potentially very dangerous. 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 measures, including those that may be used to restart the heart or breathing, may be administered when you are being infused with Lumizyme. Should treatment be discontinued, your physician may discuss restarting therapy. You may require close observation during infusions with Lumizyme administration following a severe hypersensitivity reaction.
If you or your child has an illness (i.e. a chronic breathing condition) or has compromised heart function, there may be risk of acute heart and or lung failure, and your doctor may decide that close observation during and after Lumizyme administration may be necessary.
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.
WARNING AND PRECAUTIONS
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.
Immune-Mediated Reactions: If you or your child has an illness (i.e. a chronic breathing condition) or has compromised heart function, there may be risk of acute heart and or lung failure, and your doctor may decide that close observation during and after Lumizyme administration may be necessary.
Risk of Acute Cardiorespiratory Failure: Infant Pompe patients with heart or breathing problems may be at risk for increasing the seriousness of these problems as a result of Lumizyme administration, and your child’s doctor may require additional monitoring for these infants.
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.
If you are pregnant, you should use Lumizyme only if your doctor has determined that its use outweighs any risks to your unborn child.
To report suspected adverse reactions, contact Genzyme at 1-800-745-4447 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Please see the full prescribing information for complete details, including boxed warning.