Lumizyme® (alglucosidase alfa) is a hydrolytic lysosomal glycogen-specific enzyme indicated for patients with Pompe disease (acid α-glucosidase (GAA) deficiency).
Pompe disease—also known as acid maltase deficiency or glycogen-storage disease type 2—is a progressive, debilitating, and often fatal neuromuscular disorder. It was first identified in 1932 by Johannes C. Pompe3 and has an incidence in older children and adults of about 1 in 57,000.4
Caused by a deficiency of the enzyme acid alfa glucosidase (GAA), Pompe disease is characterized by progressive glycogen accumulation primarily in the lysosomes of muscle cells. It is an autosomal recessive genetic disorder, affecting both sexes equally.
In the table below, options to determine DNA mutations or GAA activity, are listed from the least invasive to most invasive (top to bottom)
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: Since Lumizyme is a protein, it is possible that patients receiving it develop antibodies. Some patients who develop high IgG antibody levels that last for a while may have reduced response to Lumizyme. 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.
If you are pregnant, you should use Lumizyme only if your doctor has determined that its use outweighs any risks to your unborn child.
Please see the Full Prescribing Information for complete details, including boxed WARNING.