Pronunciation:

aye-GAL-si-days BAY-ta

Brand Names:

• Fabrazyme

Dosage Forms:

• Powder for Solution

Classifications:

Therapeutic—

Endocrine-Metabolic Agent

Pharmacologic—

Enzyme

Uses of This Medicine:

Agalsidase beta is used to treat Fabry disease which is an inherited disease caused by the lack of an enzyme called alpha-galactosidase A, in the body. This enzyme is necessary for your body.

Agalsidase beta 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—

Studies on this medicine have been done only in adult patients, and there is no specific information comparing use of agalsidase beta in children with use in other age groups.

Older adults—

Many medicines have not been studied specifically in older people. Therefore, it may not be known whether they work exactly the same way they do in younger adults or if they cause different side effects or problems in older people. There is no specific information comparing use of agalsidase beta in the elderly with use in other age groups.

Pregnancy—

	Pregnancy Category	Explanation
All Trimesters	B	Animal studies have revealed no evidence of harm to the fetus, however, there are no adequate studies in pregnant women OR animal studies have shown an adverse effect, but adequate studies in pregnant women have failed to demonstrate a risk to the fetus.

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 problems—May be at a higher risk of severe complications from infusion reaction.

Proper Use of This Medicine:

Dosing—

The dose of this medicine will be different for different patients. Follow your doctor's orders or the directions on the label. The following information includes only the average doses of this medicine. If your dose is different, do not change it unless your doctor tells you to do so.

The amount of medicine that you take depends on the strength of the medicine. Also, the number of doses you take each day, the time allowed between doses, and the length of time you take the medicine depend on the medical problem for which you are using the medicine.

• For Fabry disease:
  • For injection dosage form:
    • Adults—The dose is based on body weight and must be determined by your doctor. The usual dose is 1 mg per kilogram (kg) of body weight, injected slowly into a vein. The dose may be repeated once every 2 weeks.
    • Children—Use and dose must be determined by your doctor.

Missed dose—

Call your doctor or pharmacist for instructions.

Precautions While Using This Medicine:

It is important that your doctor monitor your progress while you are receiving agalsidase beta to make sure that the medicine is working properly.

It is important to take medicine for treatment of infusion reaction prior to receiving your infusion.

Importance of receiving agalsidase beta in health care facility with medical support for severe infusion reactions.

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 immediately if any of the following side effects occur:

More common

Blurred vision

chest pain or discomfort

confusion

cough producing mucus

difficulty breathing

difficulty in moving

dizziness;

fatigue

faintness or lightheadedness when getting up from a lying or sitting position suddenly

feeling unusually cold shivering

headache

hives or welts

irregular heartbeat

itching

joint pain

muscle aching or cramping

muscle pains or stiffness

nervousness

pain

pounding in the ears

redness of skin

shortness of breath

skin rash

stomach pain

sweating

swelling of ankles, feet, and lower legs

swollen joints

tightness in chest

unusual tiredness or weakness

wheezing

Incidence unknown

Cloudy or bloody urine

decreased cardiac output

difficulty in speaking

double vision

feeling of constant movement of self or surroundings

high blood pressure

inability to move arms, legs, or facial muscles

inability to speak

no blood pressure or pulse

partial loss of hearing

pounding or rapid pulse

problems with muscle control or coordination

sensation of spinning

shakiness and unsteady walk

slow speech

stopping of heart

swelling of face

swelling of the lip or ear

throat tightness

trembling

unconsciousness

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

body produces substance that can bind to drug making it less effective or cause side effects

burning, crawling, itching, numbness, prickling, "pins and needles" , or tingling feelings

congestion

discouragement

dryness or soreness of throat

fear

feeling sad or empty

fever, not related to infusion

heartburn

hoarseness

indigestion

irritability

lack of appetite

loss of interest or pleasure

nausea

pain or tenderness around eyes and cheekbones

paleness of skin

runny nose

sensation of change in temperature

skeletal pain

sneezing

stuffy nose; swelling of testes

tender, swollen glands in neck

trouble concentrating

trouble sleeping

trouble in swallowing

voice changes

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