IMPORTANT SAFETY INFORMATION:
WARNING: RISK OF ANAPHYLAXIS, HYPERSENSITIVITY AND IMMUNE-MEDIATED REACTIONS, AND RISK OF CARDIORESPIRATORY FAILURE If you or your child is taking Lumizyme, you should know that severe and potentially life-threatening allergic-type reactions known as anaphylaxis, severe hypersensitivity reactions, and reactions related to the immune system may occur during and after Lumizyme treatment. These reactions may include extreme difficulty breathing, shallow breathing, abnormal heart rate, low blood pressure, throat tightness, face and tongue swelling, kidney dysfunction, and severe skin lesions. You should seek immediate medical care if signs and symptoms of anaphylaxis, hypersensitivity reactions, and immune system reactions occur. View more

TREATMENT CENTERS

Because Pompe disease is a complex, rare neuromuscular disorder, finding a specialist with experience in treating the disorder is not always easy. However, certain treatment centers throughout the country are experienced in treating rare genetic disorders, including Pompe disease. These centers offer multidisciplinary care (including doctors that specialize in problems with the muscles, heart, or lungs, and doctors who specialize in genetics) and are active in Pompe disease research and education, and provide treatment for people living with Pompe disease. The physicians within these centers can often also consult with your own doctors, if traveling to a treatment center is not an option for you.

One helpful resource in finding treatment centers for Pompe disease is the Muscular Dystrophy Association (MDA). Pompe disease is one of more than 40 neuromuscular diseases included within the scope of the MDA’s mission and services. The organization maintains over 200 hospital-affiliated clinics with nurses, physicians, and therapists experienced in neuromuscular disorders. While the MDA does not run any of their own clinics, Pompe patients may access services through local hospitals that may be affiliated with the MDA

Please contact a CareConnectPSS Case Manager for help finding a treatment center for Pompe disease.

Indication

LUMIZYME® (alglucosidase alfa) is an enzyme replacement therapy for patients with Pompe disease (acid α-glucosidase (GAA) deficiency).

Important Safety Information

WARNING: RISK OF ANAPHYLAXIS, HYPERSENSITIVITY AND IMMUNE-MEDIATED REACTIONS, AND RISK OF CARDIORESPIRATORY FAILURE 

  • If you or your child is taking Lumizyme, you should know that severe and potentially life-threatening allergic-type reactions known as anaphylaxis, severe hypersensitivity reactions, and reactions related to the immune system may occur during and after Lumizyme treatment. These reactions may include extreme difficulty breathing, shallow breathing, abnormal heart rate, low blood pressure, throat tightness, face and tongue swelling, kidney dysfunction, and severe skin lesions. You should seek immediate medical care if signs and symptoms of anaphylaxis, hypersensitivity reactions, and immune system reactions occur. If such a reaction is severe enough, your doctor may decide to immediately discontinue the infusion and provide immediate medical care. Appropriate medical support measures may be administered during your infusion, and you may require close observation during and after Lumizyme administration.
  • If your child has compromised breathing or heart function due to Pompe disease, there may be a risk of acute worsening of heart and/or lung function with Lumizyme due to the fluid from the infusion, and your doctor may decide that close observation during and after Lumizyme administration may be necessary.

Warnings 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. 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.

Adverse Reactions

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.