Elfabrio: Uses, Side Effects and Dosage

Serious warnings and precautions box

ELFABRIO may cause hypersensitivity reactions, including severe allergic reactions during and after your infusions. ELFABRIO will be given by a healthcare professional who will monitor you closely during this time. If you experience a severe reaction, tell your healthcare professional right away. Your healthcare professional should immediately stop your infusion and start appropriate medical treatment. Your healthcare professional will then decide if you should continue your treatment with ELFABRIO. If so, your healthcare professional may try to make your body used to ELFABRIO first. To do this, they will give you small doses of ELFABRIO, gradually increasing the dose at specific intervals.

What ELFABRIO is used for:

ELFABRIO is an enzyme replacement therapy (ERT) used for the long term treatment of adults with a confirmed diagnosis of Fabry disease.

How ELFABRIO works:

Fabry disease is a rare inherited disorder that causes you to not have enough of a certain enzyme (called α-galactosidase A) that breaks down complex fats inside the cells. This results in the buildup of a specific type of fat (called globotriaosylceramide) in the cells of your body. If this buildup is not removed, it starts to accumulate in the walls of the blood vessels of your organs. This can cause a wide range of symptoms in people with Fabry disease, and even severe health complications such as heart and kidney disease.

ELFABRIO contains the active substance pegunigalsidase alfa. It replaces the missing enzyme, and helps your body break down fats inside the cells. This helps to treat some of the symptoms and prevent complications of Fabry disease.

The ingredients in ELFABRIO are:

Medicinal ingredient: pegunigalsidase alfa

Non-medicinal ingredients: citric acid, sodium chloride, sodium citrate tribasic dihydrate, and water for injection.

ELFABRIO comes in the following dosage form:

Concentrate solution for intravenous infusion; 2 mg/mL.

Do not use ELFABRIO if:

• you are allergic to pegunigalsidase alfa, or to any of the other ingredients in ELFABRIO or its container.

To help avoid side effects and ensure proper use, talk to your healthcare professional before you receive ELFABRIO. Talk about any health conditions or problems you may have, including if you:

• have had any other enzyme replacement therapies (ERTs) in the past. You may be positive for anti-drug antibodies (ADAs).
• have had an allergic reaction (no matter how severe) to ELFABRIO or had any infusion associated reactions to other ERTs in the past.
• have heart problems.
• are 65 years of age or older.
• are pregnant, think you might be pregnant or planning to become pregnant.
• are breastfeeding or planning to breastfeed.

Other warnings you should know about:

Infusion-associated reactions: They can happen at any time during and in the hours following your infusion with ELFABRIO. Symptoms may include: allergic reaction, itchy skin, feeling sick or dizzy, chills and muscle pain. Tell your healthcare professional right away if you experience any of these symptoms during this time. Depending on the severity of your symptoms, your healthcare professional may:

• temporarily stop or slow down your infusion, and give you medication. These medications are used to treat mild to moderate reactions. If your symptoms improve, your healthcare professional may continue your infusion.
• stop your infusion completely and monitor your health closely. Your healthcare professional may recommend that you take certain medications before your next infusion. This is to prevent any future severe reactions. Your healthcare professional may restart your infusion within 7 to 14 days.

Your healthcare professional may then do blood tests to determine if you have developed anti-drug antibodies (ADAs). They are produced by your body’s immune system when it identifies a drug as foreign. They can lead to the symptoms described above. If you have ADAs in your blood, this means:

• you may be at higher risk of experiencing infusion-associated reactions during your treatment with ELFABRIO; and
• it may affect ELFABRIO’s effectiveness.

Pregnancy:

• ELFABRIO is not recommended during pregnancy. If you discover that you are pregnant during your treatment with ELFABRIO, tell your healthcare professional right away.
• Pregnancy safety study: Information is being collected on patients who have been exposed or are currently exposed to ELFABRIO during pregnancy to track the effects of ELFABRIO exposure on pregnant women and their offspring. Talk to your healthcare professional for more information.

Breastfeeding: It is not known if ELFABRIO can pass into breast milk and harm a breastfed baby. ELFABRIO is not recommended during breastfeeding, unless you and your healthcare professional have determined that the potential benefits outweigh the potential risks to your child.

Kidney problems: ELFABRIO may rarely cause a condition called membranoproliferative glomerulonephritis. It is a kidney disorder that happens when your body’s immune system does not work normally. It causes a temporary decrease in kidney function. If left untreated, it may lead to kidney failure. Your healthcare professional may do blood and/or urine tests to monitor the health of your kidneys.

Driving and using machines: ELFABRIO may cause dizziness and vertigo. Do not drive, use machinery, or do activities that require you to be alert until you know how ELFABRIO affects you.

Tell your healthcare professional about all the medicines you take, including any drugs, vitamins, minerals, natural supplements or alternative medicines.

No interaction studies have been performed.

How ELFABRIO is given:

• ELFABRIO will be given to you:

  • by a healthcare professional in a healthcare setting.
  • through a drip into a vein (called an intravenous infusion) for a few hours. Your infusion time may be decreased if your healthcare professional confirms that your body is handling ELFABRIO well after the first 4 to 6 infusions. Depending on your body weight and other factors, your maintenance infusions may be as short as 1.5 hours.
• Your healthcare professional may give you other medications before you receive ELFABRIO. These include antihistamines, corticosteroids and antipyretics. These are to prevent or help relieve symptoms of an allergic reaction or other side effects associated with the infusion, such as pain, inflammation and fever. Take them exactly as directed by your healthcare professional.
• Your healthcare professional will decide if you are eligible for home infusions of ELFABRIO. If you are, you will receive an Infusion Diary with important information about possible risks and what to do if you have a reaction during your infusion. It also includes emergency contact details. Keep this diary easily accessible during your infusion. You can get extra copies if needed by contacting your healthcare professional. Home infusions must still be done under the supervision of a healthcare professional. If you experience any side effects during a home infusion with ELFABRIO, tell your healthcare professional right away. They should immediately stop your infusion and start appropriate medical treatment.

Usual dose:

• Your healthcare professional will determine the right dose for you based on your body weight.
• The usual adult dosage is 1 mg for each kg of body weight, given by intravenous infusion every other week.

Overdose:

If you think you, or a person you are caring for, have been given too much ELFABRIO, contact a healthcare professional, hospital emergency department, or regional poison control centre or Health Canada’s toll-free number, 1-844 POISON-X (1-844-764-7669) immediately, even if there are no signs or symptoms.

Missed dose:

If you have missed one or more doses, tell your healthcare professional as soon as possible to reschedule your appointment. ELFABRIO treatment should be restarted as soon as possible to maintain the 2-week interval between infusions thereafter. A double dose should not be administered to make up for a missed dose.

Possible side effects from using ELFABRIO:

These are not all the possible side effects you may have when receiving ELFABRIO. If you experience any side effects not listed here, tell your healthcare professional.

Side effects with ELFABRIO may include:

• a spinning sensation (vertigo)
• lack of energy
• nausea
• vomiting
• diarrhea
• infusion-associated reactions, which includes allergic reaction, nausea, chills, dizziness, skin rash, itching, and muscle pain
• headache
• pain, numbness, tingling or pins and needles sensations in the arms or legs
• feeling generally unwell
• chest pain
• back pain
• throat irritation
• inflammation of the sinuses
• infection of the upper respiratory tract (e.g., nose, sinuses, or throat)
• fever
• stuffy or runny nose
• cough

Serious side effects and what to do about them

If you have a troublesome symptom or side effect that is not listed here or becomes bad enough to interfere with your daily activities, tell your healthcare professional.

Reporting side effects

You can report any suspected side effects associated with the use of health products to Health Canada by:

• Visiting the Web page on Adverse Reaction Reporting (canada.ca/drug-device-reporting) for information on how to report online, by mail or by fax; or
• Calling toll-free at 1-866-234-2345.

NOTE: Contact your healthcare professional if you need information about how to manage your side effects. The Canada Vigilance Program does not provide medical advice.

Storage:

ELFABRIO will be stored by your healthcare professional as follows:

• in a refrigerator (2°C to 8°C).
• after dilution, the diluted solution should be used immediately. If not used immediately, the diluted solution should be stored for no longer than 24 hours in a refrigerator (2°C to 8°C) or for no longer than 8 hours at room temperature (below 25°C).
• do not freeze or shake.
• ELFABRIO should not be used beyond the expiration date stated on the packaging.

Keep out of reach and sight of children.

If you want more information about ELFABRIO:

• Talk to your healthcare professional
• Find the full product monograph that is prepared for healthcare professionals and includes the Patient Medication Information by visiting the Health Canada Drug Product Database website: (https://www.canada.ca/en/health-canada/services/drugs-health-products/drug-products/drug-product-database.html); the manufacturer’s website www.chiesiassist.ca; or by calling 1-833-918-4954.

Date of Authorization: December 9, 2025