Agalsidase alfa belongs to the group of medications known as enzyme replacement therapy. It is used as enzyme replacement therapy for people with Fabry disease. Fabry disease is a genetic disorder where the level of alfa-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 alfa slows the rate of progression of Fabry disease in the kidney, heart, and brain.

Agalsidase alfa has been granted a notice of compliance with conditions (NOC/c) by Health Canada. Health Canada has approved this medication to be marketed based on promising evidence of effectiveness but that additional are needed to verify optimal individual dosing. An NOC/c is used to allow access to products that are used for the treatment or prevention of serious, life-threatening or severely debilitating illness.

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 using this medication, speak to your doctor. Do not stop using this medication without consulting your doctor.

Currently, the usual dose of agalsidase alfa is 0.2 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 that this medication be given exactly as recommended by your doctor. If you miss an appointment to receive agalsidase alfa, 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 should be stored in the refrigerator. Keep this medication out of the reach of children.

Each mL of concentrated solution for infusion contains 3.5 mg of agalsidase alfa. Nonmedicinal ingredients: polysorbate 20, sodium chloride, sodium hydroxide, sodium phosphate monobasic monohydrate, and water for injection.

Do not use agalsidase alfa if you:

• are allergic to agalsidase alfa or any ingredients of the 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.

• abdominal discomfort
• acne
• change in taste of food
• changed sensitivity to touch
• decreased sense of smell
• diarrhea
• dry skin
• eye problems
• fatigue
• flushing
• headache
• heartburn
• increased blood pressure
• increased tear production
• nausea
• numbness
• "pins and needles" feeling in arms or legs
• tremors
• vomiting
• weakness

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:

• dizziness or lightheadedness
• fast, irregular, or pounding heartbeat
• fever
• infusion-related reactions (chills, fever, feeling hot or cold, high blood pressure, nausea, vomiting, flushing, burning sensation, fatigue, pain, headache, chest pain, itchy skin)
• pain or tenderness in chest, muscles, or joints
• puffiness around eyes
• sensation of spinning
• shortness of breath
• skin rash or itching
• skin redness
• swelling of the legs, feet, or hands
• symptoms of an infection (cough, runny nose, sore throat)

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

• signs of heart attack (e.g., sudden chest pain or pain radiating to back, down arm, jaw; sensation of fullness of the chest; nausea; vomiting; sweating; anxiety)

• 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.

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 alfa, 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 and fever and usually start within the first 2 to 4 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).

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 alfa passes into breast milk. If you are breast-feeding 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 this medication have not been studied for children under 7 years old.

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

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

• amiodarone
• chloroquine
• gentamicin
• hydroxychloroquine

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.