Myozyme contains an enzyme that naturally occurs in the body in healthy people. Some people lack this enzyme because of a genetic disorder. Alglucosidase alfa helps replace this missing enzyme in such people.

Myozyme is used to treat a glycogen storage disorder called Pompe disease, (also called GAA deficiency) in adults and children.

Myozyme has been shown to improve ventilator-free survival in patients with infantile-onset Pompe disease.

Some people receiving Myozyme have had serious allergic reactions during or shortly after infusion of this medicine. Tell your caregivers or get emergency medical help right away if you have any signs of a severe allergic reaction, such as itching, rash. redness or tingling, severe headache, feeling like you might pass out, fast or slow heart rate, wheezing, trouble breathing, pale skin, and blue lips or fingernails.

Before receiving Myozyme, tell your doctor if you have heart disease, lung disease or a breathing disorder, or if you are allergic to mice, hamsters, or drug products made with "murine" proteins.

Before each injection, tell your doctor if you have recently been sick with a cold, flu, or other illness. Some people receiving an injection of Myozyme have had a reaction to the infusion. This type of reaction can occur when the medicine is injected into the vein, or as long as 3 hours after the injection.

Before taking this medicine

To make sure Myozyme is safe for you, tell your doctor if you have ever had:

• heart disease; or

• lung disease or breathing disorder.

Babies who develop Pompe disease in the first year of life may need specialized medical tests to improve treatment with Myozyme treatment.

It is not known whether this medicine will harm an unborn baby. Tell your doctor if you are pregnant or plan to become pregnant.

You should not breastfeed within 24 hours after receiving Myozyme. If you use a breast pump during this time, throw out any milk you collect. Do not feed it to your baby.

Your name may be listed on the Pompe Registry. This is to track the progress of your disease and the outcome of your treatment with Myozyme.

Myozyme is given as an infusion into a vein. A healthcare provider will give you this injection.

Myozyme must be given slowly, and the IV infusion can take up to 4 hours to complete.

This medicine is usually given once every 2 weeks.

Before each injection, tell your doctor if you have recently been sick with a cold, flu, or other illness.

If you need surgery, tell the surgeon ahead of time that you are using Myozyme.

Myozyme can have long lasting effects on your body. Your blood will need to be tested every 3 months for 2 years and then once every year after that.

Usual Adult Dose for Pompe disease:

20 mg/kg via IV infusion every 2 weeks

Comments:
-Total volume of infusion is determined by patient's body weight and should be infused over about 4 hours.

Usual Pediatric Dose for Pompe disease:

20 mg/kg via IV infusion every 2 weeks

Comments:
-Total volume of infusion is determined by body weight and should be infused over about 4 hours.

To make sure Myozyme is safe for you, tell your doctor if you have ever had:

• heart disease; or

• lung disease or breathing disorder.

Babies who develop Pompe disease in the first year of life may need specialized medical tests to improve treatment with Myozyme treatment.

It is not known whether this medicine will harm an unborn baby. Tell your doctor if you are pregnant or plan to become pregnant.

You should not breastfeed within 24 hours after receiving Myozyme. If you use a breast pump during this time, throw out any milk you collect. Do not feed it to your baby.

Your name may be listed on the Pompe Registry. This is to track the progress of your disease and the outcome of your treatment with Myozyme.

Call your doctor for instructions if you miss an appointment for your Myozyme injection.

Seek emergency medical attention or call the Poison Help line at 1-800-222-1222.

Follow your doctor's instructions about any restrictions on food, beverages, or activity.

Get emergency medical help if you have signs of an allergic reaction to Myozyme: hives; difficult breathing; swelling of your face, lips, tongue, or throat.

Some allergic reactions may occur during the injection, or as long as 3 hours afterward. Tell your caregivers or get emergency medical help right away if you have:

• a feeling like you might pass out, even while lying down;

• slow heart rate, weak pulse, fainting, slow breathing (breathing may stop);

• bronchospasm (wheezing, tight feeling in your chest or throat, trouble breathing);

• pale skin, cold or clammy skin, blue lips or fingernails, cold hands or feet;

• warmth, redness, numbness, or tingly feeling under your skin;

• rash, itching, sweating, fever;

• severe headache, nausea, feeling restless or nervous;

• seizure (convulsions); or

• fast heart rate, pounding in your neck or ears, anxiety, confusion.

Also call your doctor at once if you have:

• skin changes (sores, lesions, ulcers, discoloring); or

• puffy eyes, swelling in your ankles or feet, weight gain, urine that looks foamy.

Common Myozyme side effects may include:

• hives; difficult breathing; swelling of your face, lips, tongue, or throat;

• pale skin, blue lips, feeling hot or feverish;

• skin rash, itching, flushing (warmth, redness, or tingly feeling);

• headache, dizziness, agitation, muscle twitching;

• fast heart rate, rapid breathing, chest discomfort, cough;

• increased blood pressure;

• nausea, vomiting;

• tremor, shaking, increased sweating; or

• muscle pain, tiredness.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.