How does this medication work? What will it do for me?

Agalsidase beta belongs to the group of medications known as enzyme replacement therapy. It is used as enzyme replacement therapy for people with Fabry disease, which is a genetic disorder where the levels of alpha-galactosidase (an enzyme that breaks down fats) is low or absent. With Fabry disease, globotriaosylceramide (or GL-3; a fat substance) is not removed from cells in the body and starts to accumulate in the walls of blood vessels and organs. By reducing GL-3 levels in the body, agalsidase beta slows the rate of progression of Fabry disease in the kidney, heart, and brain.

Your doctor may have suggested this medication for conditions other than those listed in these drug information articles. As well, some forms of this medication may not be used for all of the conditions discussed here. If you have not discussed this with your doctor or are not sure why you are being given this medication, speak to your doctor. Do not stop using this medication without consulting your doctor.

Do not give this medication to anyone else, even if they have the same symptoms as you do. It can be harmful for people to take this medication if their doctor has not prescribed it.

What form(s) does this medication come in?

5 mg
Each vial of sterile, nonpyrogenic, white to off-white lyophilized cake or powder, contains agalsidase beta 5.5 mg total amount, which allows for an extractable dose of 5 mg/vial. Nonmedicinal ingredients: mannitol, sodium phosphate monobasic, monohydrate, sodium phosphate dibasic, and heptahydrate. This medication does not contain any preservatives.

35 mg
Each vial of sterile, nonpyrogenic, white to off-white lyophilized cake or powder, contains agalsidase beta 37 mg total amount, which allows for an extractable dose of 35 mg/vial. Nonmedicinal ingredients: mannitol, sodium phosphate monobasic, monohydrate, sodium phosphate dibasic, and heptahydrate. This medication does not contain any preservatives.

How should I use this medication?

The usual dose of agalsidase beta is 1 mg per kilogram of body weight given every 2 weeks as an infusion into a vein.

Many things can affect the dose of medication that a person needs, such as body weight, other medical conditions, and other medications. If your doctor has recommended a dose different from the ones listed here, do not change the way that you are using the medication without consulting your doctor.

It is important this medication be given exactly as recommended by your doctor. If you miss an appointment to receive agalsidase beta, contact your doctor as soon as possible to reschedule your appointment. If you are receiving this medication at home and miss a dose, contact your doctor.

Vials of this medication are stored in the refrigerator. After prepared for an infusion, this medication should be used immediately.

Do not dispose of medications in wastewater (e.g. down the sink or in the toilet) or in household garbage. Ask your pharmacist how to dispose of medications that are no longer needed or have expired.

Who should NOT take this medication?

Do not use this medication if you are allergic to agalsidase beta or any ingredients of the medication.

What side effects are possible with this medication?

Many medications can cause side effects. A side effect is an unwanted response to a medication when it is taken in normal doses. Side effects can be mild or severe, temporary or permanent.

The side effects listed below are not experienced by everyone who takes this medication. If you are concerned about side effects, discuss the risks and benefits of this medication with your doctor.

The following side effects have been reported by at least 1% of people taking this medication. Many of these side effects can be managed, and some may go away on their own over time.

Contact your doctor if you experience these side effects and they are severe or bothersome. Your pharmacist may be able to advise you on managing side effects.

  • anxiety
  • back pain
  • decreased hearing
  • depression
  • difficulty sleeping
  • pain, including muscle and neck pain
  • ringing in the ears
  • skin rash
  • stomach discomfort
  • symptoms of an infection (cough, runny nose, sore throat)
  • toothache

Although most of the side effects listed below don't happen very often, they could lead to serious problems if you do not seek medical attention.

Check with your doctor as soon as possible if any of the following side effects occur:

  • chest tightness
  • dizziness or lightheadedness
  • fast heartbeat
  • infusion-related reactions (e.g., chills, fever, feeling hot or cold, high blood pressure, nausea, vomiting, flushing, burning sensation, fatigue, pain, headache, chest pain, itchy skin)
  • signs of depression (e.g., poor concentration, changes in weight, changes in sleep, decreased interest in activities, thoughts of suicide)
  • skin abrasions due to skin scratching
  • swelling of the legs, feet, or hands
  • symptoms of anemia (e.g., paleness, shortness of breath, fast heartbeat)
  • throat tightness

Stop taking the medication and seek immediate medical attention if any of the following occur:

  • symptoms of a serious allergic reaction (e.g., shortness of breath; difficulty breathing; wheezing; hives; swelling of the face, lips, or throat)

Some people may experience side effects other than those listed. Check with your doctor if you notice any symptom that worries you while you are taking this medication.

Are there any other precautions or warnings for this medication?

Before you begin using a medication, be sure to inform your doctor of any medical conditions or allergies you may have, any medications you are taking, whether you are pregnant or breast-feeding, and any other significant facts about your health. These factors may affect how you should use this medication.

Allergic and infusion-related reactions: This medication can cause allergic reactions, some of which can be severe. You will receive this medication under close medical supervision, especially at the beginning of treatment.

Most people develop antibodies when treated with enzyme replacement therapy. If you develop antibodies to agalsidase beta, you might experience allergic side effects such as an infusion-related reaction. The antibodies are not likely to stop this medication from working and will decrease with time.

Infusion-related reactions can include chills, fever, increased blood pressure, feeling hot or cold, burning sensations, nausea, vomiting, flushing, fatigue, pain, headache, chest pain, or itchy skin, and usually within the first 3 months of treatment. If you experience an infusion-related reaction, your doctor can decrease the infusion rate or treat the reaction with other medications (e.g., acetaminophen, ibuprofen, antihistamines, or corticosteroids).

Heart rhythm: Agalsidase beta can cause changes to the normal rhythm of the heart, including an irregular heartbeat called QT prolongation. QT prolongation is a serious life-threatening condition that can cause fainting, seizures, and sudden death.

If you are at risk for heart rhythm problems (e.g., people with heart failure, angina, low potassium or magnesium levels), discuss with your doctor how this medication may affect your medical condition, how your medical condition may affect the dosing and effectiveness of this medication, and whether any special monitoring is needed.

Pregnancy: This medication should not be used during pregnancy unless the benefits outweigh the risks. If you become pregnant while taking this medication, contact your doctor immediately.

Breast-feeding: It is not known if agalsidase beta passes into breast milk. If you are a breast-feeding mother and are taking this medication, it may affect your baby. Talk to your doctor about whether you should continue breast-feeding.

Children: The safety and effectiveness of using this medication have not been established for children under 8 years of age.

Seniors: The safety and effectiveness of using this medication have not been established for seniors.

What other drugs could interact with this medication?

There may be an interaction between agalsidase beta and any of the following:

  • amiodarone
  • chloroquine
  • gentamicin
  • monobenzone

If you are taking any of these medications, speak with your doctor or pharmacist. Depending on your specific circumstances, your doctor may want you to:

  • stop taking one of the medications,
  • change one of the medications to another,
  • change how you are taking one or both of the medications, or
  • leave everything as is.

An interaction between two medications does not always mean that you must stop taking one of them. Speak to your doctor about how any drug interactions are being managed or should be managed.

Medications other than those listed above may interact with this medication. Tell your doctor or prescriber about all prescription, over-the-counter (non-prescription), and herbal medications you are taking. Also tell them about any supplements you take. Since caffeine, alcohol, the nicotine from cigarettes, or street drugs can affect the action of many medications, you should let your prescriber know if you use them.