alglucosidase alfa (injection)

What is the most important information I should know about alglucosidase alfa?

Alglucosidase alfa is available only under a special program called ACE. Under this program, only registered doctors and pharmacists can prescribe and dispense alglucosidase alfa. You must be registered in the program and sign documents stating that you understand the risks of using this medication and the possibility of severe allergic reaction. Ask your doctor or call the drug maker if you have questions about the program or the written requirements.

Before receiving this medication, 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 alglucosidase alfa 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.

Tell your caregivers or get emergency medical help right away if you have any signs of a severe allergic reaction, such as feeling restless, nervous, dizzy, numb, tingly, hot or cold, sweaty, nauseated, or lightheaded, or if you have trouble breathing, chest pain or tightness, fast or slow heart rate, hives, severe skin rash, seizure (convulsions), or swelling of your face, lips, tongue, or throat.

What is alglucosidase alfa?

Alglucosidase alfa 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.

Alglucosidase alfa is used to treat a glycogen storage disorder called Pompe disease, (also called GAA deficiency) in adults and children who are at least 8 years old.

Alglucosidase alfa may also be used for purposes not listed in this medication guide.

What should I discuss with my health care provider before receiving alglucosidase alfa?

You should not receive alglucosidase alfa if you are allergic to it.

If you have any of these other conditions, you may need a dose adjustment or special tests:

• heart disease;
• lung disease or breathing disorder;
• if you are allergic to mice, hamsters, or drug products made with "murine" proteins.

FDA pregnancy category B. Alglucosidase alfa is not expected to harm an unborn baby. Tell your doctor if you are pregnant or plan to become pregnant during treatment.

It is not known whether alglucosidase alfa passes into breast milk or if it could harm a nursing baby. Do not receive this medication without telling your doctor if you are breast-feeding a 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 alglucosidase alfa.

How is alglucosidase alfa given?

Alglucosidase alfa is injected into a vein through an IV using an infusion pump. You will receive this injection in a clinic or hospital setting. Alglucosidase alfa must be given slowly, and the IV infusion can take up to 4 hours to complete.

This medication 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.

To be sure this medicine is helping your condition and is not causing harmful effects, your blood will need to be tested every 3 months for 2 years and then once every year after that. Visit your doctor regularly.

Alglucosidase alfa is available only under a special program called ACE. Under this program, only registered doctors and pharmacists can prescribe and dispense alglucosidase alfa. You must be registered in the program and sign documents stating that you understand the risks of using this medication and the possibility of severe allergic reaction. Ask your doctor or call the drug maker if you have questions about the program or the written requirements.

What happens if I miss a dose?

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

What happens if I overdose?

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

What should I avoid while receiving alglucosidase alfa?

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

What are the possible side effects of alglucosidase alfa?

Some people receiving an injection of alglucosidase alfa 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. Tell your caregivers or get emergency medical help right away if you have any of these signs of a severe allergic reaction:

• feeling like you might pass out, even while lying down;
• feeling restless, nervous, dizzy, or nauseated;
• pale skin, redness under your skin, sweating, feeling hot or cold;
• fast or slow heart rate;
• pain or tightness in your chest or throat;
• wheezing, trouble breathing;
• cold hands, blue lips;
• back pain;
• numbness, warmth, redness, or tingly feeling;
• seizure (convulsions);
• hives, severe skin rash; or
• swelling of your face, lips, tongue, or throat.

Call your doctor at once if you have a serious side effect such as:

• pain or fullness in your ear, problems with hearing;
• skin ulcers;
• fast, slow, or uneven heartbeats;
• weak pulse, fainting, slow breathing (breathing may stop); or
• chest pain or heavy feeling, pain spreading to the arm or shoulder, sweating, general ill feeling.

Less serious side effects may include:

• mild skin rash or itching;
• diarrhea, constipation, upset stomach, vomiting;
• sore throat, neck pain;
• pain or swelling in your arms or legs; or
• pain, swelling, burning, or irritation around the IV needle.

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.

What other drugs will affect alglucosidase alfa?

There may be other drugs that can interact with alglucosidase alfa. Tell your doctor about all medications you use. This includes prescription, over-the-counter, vitamin, and herbal products. Do not start a new medication without telling your doctor.

Where can I get more information?

Your doctor or pharmacist can provide more information about alglucosidase alfa.