Lumizyme

Lumizyme is a prescription medication used to treat patients with Pompe disease.

Lumizyme belongs to a group of drugs called a lysosomal glycogen-specific enzyme. These work by replacing the deficient GAA, which is seen in Pompe disease, thereby reducing the accumulated glycogen in heart and skeletal muscle cells.

This medication is available in an injectable form to be given directly into a vein (IV) by a healthcare professional.

Common side effects of Lumizyme include infusion-related reactions and severe allergic reactions.

Lumizyme is a prescription medication used to treat patients with Pompe disease.

This medication may be prescribed for other uses. Ask your doctor or pharmacist for more information.

• Genzyme Corporation

Serious side effects have been reported with Lumizyme. See the “Lumizyme Precautions” section.

Common side effects of Lumizyme include the following:

• infusion-related reactions
• severe allergic reactions
• hives
• diarrhea
• vomiting
• shortness of breath
• itchy skin
• skin rash
• redness of skin
• neck pain
• partial hearing loss
• flushing/feeling hot
• pain in extremities
• chest discomfort
• pneumonia
• respiratory failure
• respiratory distress
• respiratory syncytial virus infection
• gastroenteritis
• fever
• sore throat

This is not a complete list of Lumizyme side effects. Ask your doctor or pharmacist for more information.

Tell your doctor if you have any side effect that bothers you or that does not go away.

Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.

No drug interactions have been studied by the manufacturer. However, you should tell your doctor about all the medicines you take including prescription and non-prescription medicines, vitamins, and herbal supplements. Not all drug interactions are known or reported and new drug interactions are continually being reported.

Serious side effects have been reported with Lumizyme including the following:

Anaphylaxis and hypersensitivity reactions. Serious allergic reactions can occur with Lumizyme infusion. These reactions can occur up to 3 hours after you receive your dose of Lumizyme. Tell your healthcare provider immediately if you experience any of the following signs or symptoms:

• swelling of the face, eyes, lips, tongue, arms, or legs
• difficulty breathing or swallowing
• rash
• low blood pressure

Immune-mediated reactions. Severe skin-related and other immune system-related reactions can happen within several weeks to 3 years after receiving treatment with Lumizyme. Your doctor will monitor you for the development of reactions involving your skin and other organs. If these reactions occur, your doctor may decide to discontinue the infusion.

Risk of acute cardiorespiratory failure. If you have a condition that affects your heart or lung function, Lumizyme infusion can cause an acute worsening of these problems. Your doctor may decide to closely monitor you during and after a Lumizyme infusion.

Risk of antibody development. Most patients receiving Lumizyme develop IgG antibodies, which are normally produced by the body in response to things it does not recognize as naturally being a part of your body, within the first 3 months of treatment. If the levels of these antibodies stay high for a long period of time, Lumizyme may not work as well. Your doctor should do a blood test to check your level of IgG antibodies once every 3 months for the first 2 years of treatment with Lumizyme, and then once a year thereafter.

Do not receive Lumizyme if you are allergic to Lumizyme or to any of its ingredients.

Medications can interact with certain foods. In some cases, this may be harmful and your doctor may advise you to avoid certain foods. In the case of Lumizyme, there are no specific foods that you must exclude from your diet when receiving this medication.

Before taking Lumizyme, tell your doctor about all of your medical conditions. Especially tell your doctor if you:

• are allergic to Lumizyme or to any of its ingredients
• have heart problems
• have lung problems
• are pregnant or plan to become pregnant
• are breastfeeding or plan to breastfeed

Tell your doctor about all the medicines you take including prescription and non-prescription medicines, vitamins, and herbal supplements.

Tell your doctor if you are pregnant or plan to become pregnant.

The FDA categorizes medications based on safety for use during pregnancy. Five categories - A, B, C, D, and X, are used to classify the possible risks to an unborn baby when a medication is taken during pregnancy.

Tell your doctor if you are breastfeeding or plan to breastfeed.

Lumizyme has been detected in human milk for up to 24 hours after receiving an infusion of the medication. A breastfeeding woman may temporarily pump and discard her breast milk made during the 24 hours after receiving Lumizyme. Caution should be used when choosing to administer Lumizyme to a nursing mother.

Lumizyme comes in injectable form to be given directly into a vein (IV) by a healthcare provider. Lumizyme is infused once every 2 weeks. The infusion should be around 4 hours, but may change depending on how you tolerate this medication.

If you miss an appointment to receive a dose of Lumizyme, contact your healthcare provider immediately to discuss a plan to receive your next dose.

The dose of Lumizyme your doctor recommends will be based on your weight.

The recommended dose of Lumizyme is 20 mg per kg of body weight given once every 2 weeks as an intravenous (IV) infusion.

If you have reactions to a Lumizyme infusion, your doctor may slow the infusion or stop it altogether.

Since Lumizyme is administered by a healthcare provider in a medical setting, it is unlikely that an overdose will occur. However, if overdose is suspected, seek emergency medical attention.

• It is important to keep all appointments to receive doses of Lumizyme.
• Talk to your doctor about the registry for patients with Pompe disease.

WARNING: ANAPHYLAXIS

Life-threatening anaphylactic reactions, severe allergic reactions and immune mediated reactions have been observed in some patients during Lumizyme infusions. Therefore, appropriate medical support should be readily available when Lumizyme is administered.

Infantile-onset Pompe disease patients with compromised cardiac or respiratory function may be at risk of serious acute exacerbation of their cardiac or respiratory compromise due to fluid overload, and require additional monitoring.