Pronunciation:

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

Brand Names:

• Lumizyme
• Myozyme

Dosage Forms:

• Powder for Solution

Warnings:

Life-threatening anaphylactic reactions and severe hypersensitivity reactions, presenting as respiratory distress, hypoxia, apnea, dyspnea, bradycardia, tachycardia, bronchospasm, throat tightness, hypotension, angioedema (including tongue or lip swelling, periorbital edema, and face edema), and urticaria, have occurred in some patients during and after alglucosidase alfa infusions. Immune-mediated reactions presenting as proteinuria, nephrotic syndrome, and necrotizing skin lesions have occurred in some patients following alglucosidase alfa treatment. Closely observe patients during and after alglucosidase alfa administration and be prepared to manage anaphylaxis and hypersensitivity reactions. Inform patients of the signs and symptoms of anaphylaxis, hypersensitivity reactions, and immune-mediated reactions and have them seek immediate medical care should signs and symptoms occur. 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 that treats infantile-onset Pompe disease, which is also called glycogen storage disease type II. Alglucosidase alfa contains a human enzyme called acid alpha-glucosidase. This enzyme helps with the digestion and absorption of glycogen. People with Pompe disease are not able to make enough of this enzyme.

This medicine is available only with your doctor's prescription.

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—

No information is available on the relationship of age to the effects of alglucosidase alfa injection in the geriatric population.

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:

• Breathing problems or
• Cardiac hypertrophy (heart is larger than normal) or
• Heart rhythm problems (eg, arrhythmia) or
• Lung disease—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 a needle placed in one of your veins.

This medicine must be given slowly, so the needle will remain in place for a few hours. You or your child may also receive medicines to help prevent possible allergic reactions to the injection.

Precautions While Using This Medicine:

It is very important that your doctor check the progress of you or your child at regular visits to make sure that this medicine is working properly. Blood tests may be needed to check for unwanted effects.

This medicine may cause chest pain, fever, chills, itching, hives or a rash, a fast heartbeat, flushing of the face, dizziness, fainting, or lightheadedness, trouble breathing, or swelling of the face, tongue, and throat within a few hours after it is given. Check with your doctor or nurse right away if you or your child have any of these symptoms.

This medicine may cause serious types of allergic reactions, including anaphylaxis. Anaphylaxis can be life-threatening and requires immediate medical attention. Call your doctor right away if you or your child have a rash, itching, hoarseness, lightheadedness, dizziness, or fainting, trouble breathing, trouble swallowing, or any swelling of your hands, face, or mouth after receiving this medicine.

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

chest discomfort or pain

chills

cough

difficult or labored breathing

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

nasal congestion

pain

pale skin

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

rapid shallow breathing

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

tightness in the chest

trembling or shaking of the hands or feet

troubled breathing

troubled breathing with exertion

unusual bleeding or bruising

unusual tiredness or weakness

Less common

Blood in the urine

convulsions

decreased urine

dry mouth

increased thirst

loss of appetite

mood changes

muscle pain or cramps

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

Incidence not known

Blue-green to black skin discoloration

blurred vision

confusion

dilated neck veins

extreme fatigue

heart stops

inability to speak

no breathing

no pulse or blood pressure

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

seizures

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

Abdominal or stomach pain

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

muscle or bone pain, stiffness, or tightness

muscle twitching

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

welts

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

muscle spasm

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.