Myozyme treats Pompe disease, a rare, inherited and often fatal disorder that disables the heart and skeletal muscles. Ask your doctor about the Pompe Registry.
Myozyme is a prescription medication used to treat patients with Pompe disease, a rare, inherited disorder caused by the buildup of a complex sugar called glycogen in the body's cells. The accumulation of glycogen in certain organs and tissues, especially muscles, impairs their ability to function normally.
Myozyme 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 Myozyme include infusion-related reactions and severe allergic reactions.
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Uses of Myozyme
Myozyme is a prescription medication used to treat patients with Pompe disease. Myozyme has been shown to improve ventilator-free survival in patients with infantile-onset Pompe disease.
This medication may be prescribed for other uses. Ask your doctor or pharmacist for more information.
Side Effects of Myozyme
Serious side effects have been reported with Myozyme. See the “Myozyme Precautions” section.
Common side effects of Myozyme include the following:
- infusion-related reactions
- severe allergic reactions
- shortness of breath
- itchy skin
- skin rash
- redness of skin
- neck pain
- partial hearing loss
- flushing/feeling hot
- pain in extremities
- chest discomfort
- respiratory failure
- respiratory distress
- respiratory syncytial virus infection
- sore throat
This is not a complete list of Myozyme 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 Myozyme including the following:
Anaphylaxis and allergic reactions. Severe allergic reactions have been reported in some patients during a Myozyme infusion and for up to 3 hours after it is complete. Tell your healthcare provider about any signs or symptoms of an allergic reaction, which include the following:
- chest pain
- swelling of the eyes, face, lips, tongue, arms, or legs
- difficulty breathing or swallowing
Severe skin reactions. Your immune system may develop certain proteins called antibodies that can cause severe reactions to Myozyme. These reactions have occurred several weeks to 3 years after receiving Myozyme. Your doctor should monitor you for development of any skin or other reactions to Myozyme. Tell your doctor or get medical help right away if you get any of these symptoms at anytime during your treatment:
- painful sores or ulcers on your skin, lips, or in your mouth
- peeling skin
Risk of acute cardiorespiratory failure. People with heart or lung problems may be at risk for a serious acute worsening of their condition when receiving Myozyme and for up to 72 hours after the infusion is complete. Tell your doctor if you have any conditions that affect your heart or lungs.
Infusion reactions. Infusion reactions can occur with this medication. Tell your doctor or get medical help right away if you get any of these symptoms during or after an infusion this medication:
- hives (red itchy welts) or rash
- swelling of your lips, tongue, throat or face
- sudden cough
- shortness of breath, difficulty breathing, or wheezing
- dizziness or feel faint
- palpitations (feel like your heart is racing or fluttering)
- chest pain
Antibody formation. You can develop IgG antibodies (proteins that your immune system produces in response to something it does not recognize as a normal part of your body) in response to Myozyme that increase your risk for allergic and infusion reactions to Myozyme. Your doctor will perform blood tests to monitor for development of these antibodies once every 3 months for 2 years and then once a year thereafter.
Do not take Myozyme if you are allergic to Myozyme or to any of its ingredients.
Myozyme Food Interactions
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 Myozyme, there are no specific foods that you must exclude from your diet when receiving this medication.
Before taking Myozyme, tell your doctor about all of your medical conditions. Especially tell your doctor if you:
- are allergic to Myozyme or to any of its ingredients
- have heart problems
- have lung or breathing 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.
Myozyme and Pregnancy
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.
Myozyme falls into category B. There are no well-done studies that have been done in humans with Myozyme. In animal studies, pregnant animals were given this medication, and the babies did not show any medical issues related to this medication. Myozyme should only be used in pregnant women if clearly needed.
A registry has been created to better understand Pompe disease and evaluate the long-term effects of Myozyme. The registry also monitors the effects of Myozyme on pregnant women and their offspring. For more information on joining this registry, talk to your doctor or visit www.pomperegistry.com.
Myozyme and Lactation
Tell your doctor if you are breastfeeding or plan to breastfeed.
It is not known if Myozyme crosses into human milk. Because many medications can cross into human milk and because of the possibility for serious adverse reactions in nursing infants with use of this medication, a choice should be made whether to stop nursing or stop the use of this medication. Your doctor and you will decide if the benefits outweigh the risk of using Myozyme.
Myozyme is available as an injection to be given directly into a vein (IV) by a healthcare professional, given once every 2 weeks.
The infusion is given over approximately 4 hours.
If you miss an appointment to receive an infusion of Myozyme, contact your doctor to make plans to receive your next dose.
The dose your doctor recommends will be based on your weight.
The recommended dose of Myozyme is 20 mg per kg body weight given once every 2 weeks as an intravenous (IV) infusion.
If Myozyme 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.
- Keep all appointments with your doctor and laboratory.
A registry has been created to better understand Pompe disease and evaluate the long-term effects of Myozyme. For more information on joining this registry, talk to your doctor or visit www.pomperegistry.com.
Myozyme FDA Warning
Life-threatening anaphylactic reactions, severe allergic reactions and immune mediated reactions have been observed in some patients during Myozyme infusions. Therefore, appropriate medical support should be readily available when Myozyme is administered.
Warning: CARDIORESPIRATORY FAILURE:
Risk of Cardiorespiratory Failure patients with compromised cardiac or respiratory function may be at risk of serious acute exacerbation of their cardiac or respiratory compromise due to infusion reactions, and require additional monitoring.