Pronunciation:

al-gloo-KOE-si-dase AL-fa

Brand Names:

• Lumizyme
• Myozyme

Dosage Forms:

• Powder for Solution

Warnings:

Patients treated with alglucosidase alfa have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Appropriate medical monitoring and support measures, including cardiopulmonary resuscitation equipment, should be readily available during alglucosidase alfa administration. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue alglucosidase alfa immediately and initiate appropriate medical treatment. Immune-mediated reactions presenting as proteinuria, nephrotic syndrome, and necrotizing skin lesions have occurred in some patients following alglucosidase alfa treatment. Monitor patients for the development of systemic immune-mediated reactions involving skin and other organs while receiving alglucosidase alfa. 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 .

Classifications:

Therapeutic—

Enzyme Replacement

Pharmacologic—

Enzyme

Uses of This Medicine:

Alglucosidase alfa injection is an enzyme used to treat Pompe disease (glycogen storage disease type II), including infantile-onset Pompe disease. This medicine contains a human enzyme called acid alpha-glucosidase, which helps with the digestion and absorption of glycogen. People with Pompe disease are not able to make enough of this enzyme.

This medicine is to be given only by or under the direct supervision of your doctor.

Before Using This Medicine:

In deciding to use a medicine, the risks of taking the medicine must be weighed against the good it will do. This is a decision you and your doctor will make. For this medicine, the following should be considered:

Allergies—

Tell your doctor if you have ever had any unusual or allergic reaction to this medicine or any other medicines. Also tell your health care professional if you have any other types of allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.

Children—

Appropriate studies performed to date have not demonstrated pediatric-specific problems that would limit the usefulness of alglucosidase alfa injection in children.

Older adults—

Appropriate studies performed to date have not demonstrated geriatric-specific problems that would limit the usefulness of alglucosidase alfa injection in the elderly.

Breast-feeding—

There are no adequate studies in women for determining infant risk when using this medication during breastfeeding. Weigh the potential benefits against the potential risks before taking this medication while breastfeeding.

Other medicines—

Although certain medicines should not be used together at all, in other cases two different medicines may be used together even if an interaction might occur. In these cases, your doctor may want to change the dose, or other precautions may be necessary. Tell your healthcare professional if you are taking any other prescription or nonprescription (over-the-counter [OTC]) medicine.

Other interactions—

Certain medicines should not be used at or around the time of eating food or eating certain types of food since interactions may occur. Using alcohol or tobacco with certain medicines may also cause interactions to occur. Discuss with your healthcare professional the use of your medicine with food, alcohol, or tobacco.

Other medical problems—

The presence of other medical problems may affect the use of this medicine. Make sure you tell your doctor if you have any other medical problems, especially:

• Heart disease or
• Heart rhythm problems (eg, arrhythmia) or
• Lung or breathing problems—Use with caution. May make these conditions worse.

Proper Use of This Medicine:

A nurse or other trained health professional will give you or your child this medicine in a hospital. This medicine is given through an IV catheter that is placed in one of your veins.

The medicine must be injected slowly, so the IV will need to stay in place for 4 hours. It is usually given every 2 weeks.

Your doctor will give you other medicines (eg, allergy, fever, or steroid medicine) before you receive this medicine to help prevent unwanted effects.

Missed dose—

Call your doctor or pharmacist for instructions.

Precautions While Using This Medicine:

It is very important that your doctor check your or your child's progress closely at regular visits to make sure this medicine is working properly and to decide if you should continue to receive it. Blood and urine tests may be needed to check for unwanted effects.

This medicine may cause serious allergic reactions, including anaphylaxis, which can be life-threatening and require immediate medical attention. Tell your doctor right away if you or your child have blurred vision, chest discomfort or tightness, confusion, dizziness, faintness, or lightheadedness when getting up suddenly from a lying or sitting position, fast heartbeat, flushing, redness of the skin, rash, itching, hoarseness, sweating, trouble breathing, trouble swallowing, unusually warm skin, any swelling of the hands, face, or mouth, or unusual tiredness or weakness after receiving this medicine.

This medicine may cause infusion-related reactions, which can be life-threatening and require immediate medical attention. Tell your doctor right away if you or your child start to have chest discomfort, fever, chills or shaking, confusion, difficulty swallowing, dizziness, fast heartbeat, flushing, redness of the skin, increased or decreased blood pressure, trouble breathing, itching or rash, unusually warm skin, or lightheadedness, or fainting after receiving this medicine.

Immune-mediated reactions (eg, protein in the urine, kidney problems, and skin reactions) may occur while you or your child are receiving this medicine. Check with your doctor away if you or your child have bumps and rash on the skin, cloudy or bloody urine, high blood pressure, or swelling of the face, feet, or lower legs.

Side Effects of This Medicine:

Along with its needed effects, a medicine may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor or nurse immediately if any of the following side effects occur:

More common

Blue lips, fingernails, or skin

body aches or pain

bone pain

chest discomfort, pain, or tightness

chills

cough

difficulty with swallowing

dizziness

dry, red, hot, or irritated skin

ear congestion

fast, pounding, or irregular heartbeat or pulse

feeling of warmth

fever

headache

hives, itching, or skin rash

increased sweating

irregular, fast, slow, or shallow breathing

lightheadedness, dizziness, or fainting

loss of voice

muscle pain, cramps, spasm, stiffness, tightness, or twitching

nasal congestion

pain

pale skin

puffiness or swelling of the eyelids or around the eyes, face, lips, or tongue

redness of the face, neck, arms, and occasionally, upper chest

redness or pain at the catheter site

runny nose

shakiness in the legs, arms, hands, or feet

slow or irregular heartbeat

sneezing

sore throat

swollen, painful, or tender lymph glands in the neck, armpit, or groin

trembling or shaking of the hands or feet

trouble breathing

unusual bleeding or bruising

unusual tiredness or weakness

Less common

Blood in the urine

decreased urine

dry mouth

increased thirst

loss of appetite

mood changes

nausea or vomiting

numbness or tingling in the hands, feet, or lips

pain in the groin or genitals

sharp back pain just below the ribs

seizures

Incidence not known

Blue-green to black skin discoloration

blurred vision

bumps and rash on the skin

cloudy urine

confusion

dilated neck veins

extreme fatigue

heart stops

high blood pressure

inability to speak

no breathing

no pulse or blood pressure

pain, redness, swelling, or sloughing of the skin at the place of injection

severe or sudden headache

severe pain in the chest

slurred speech

sudden and severe weakness in the arm or leg on one side of the body

sudden onset of severe breathing difficulty

swelling of the face, fingers, feet, or lower legs

temporary blindness

unconscious

weight gain

Some side effects may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:

More common

Acid or sour stomach

belching

body aches or pain

change in hearing

congestion

constipation

dryness or soreness of the throat

ear discomfort or pain

ear drainage

earache

feeling of constant movement of self or surroundings

general feeling of discomfort or illness

heartburn

hoarseness

indigestion

redness of the skin

redness or swelling in the ear

sensation of spinning

sore mouth or tongue

stomach discomfort, upset, or pain

tender, swollen glands in the neck

upper abdominal or stomach pain

voice changes

white patches in the mouth or on the tongue

Less common

Bloody nose

sleepiness or unusual drowsiness

Incidence not known

Burning, dry, or itching eyes

discharge, excessive tearing

redness, pain, or swelling of the eye, eyelid, or inner lining of the eyelid

Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.

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