Elfabrio 2 mg/ml concentrado para solucion para perfusion

Summary of Product Characteristics: Information for the User

Elfabrio 2 mg/ml Concentrate for Solution for Infusion

pegunigalsidasa alfa

This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. You can help by reporting any side effects you get. The final part of section 4 includes information on how to report these side effects.

Read this leaflet carefully before you start using this medicine because it contains important information for you.

• Keep this leaflet, as you may need to read it again.
• If you have any further questions, ask your doctor or pharmacist.
• This medicine has been prescribed for you only. Do not pass it on to others even if their symptoms are the same as yours. It may harm them.
• If you get any side effects, talk to your doctor or pharmacist. This includes any possible side effects not listed in this leaflet. See section 4.

Elfabrio contains the active ingredient pegunigalsidase alfa, and is used as an enzyme replacement therapy in adult patients with confirmed Fabry disease. Fabry disease is a rare genetic disorder that can affect many parts of the body. In patients with Fabry disease, a type of fat is not removed from the body's cells and accumulates in the walls of blood vessels, which can cause organ failure. This fat accumulates in the cells of these patients because they do not have a sufficient amount of an enzyme called α- galactosidase-A, the enzyme responsible for breaking it down.Elfabrio is used long-term to supplement or replace this enzyme in adult patients who have confirmed Fabry disease.

Do not use Elfabrio

• if you are very allergic to pegunigalsidasa alfa or any of the other components of this medication (listed in section 6).

Warnings and precautions

Consult your doctor before starting to use Elfabrio.

If you receive treatment with Elfabrio, you may experience an adverse effect during or immediately after administration of the medication via infusion pump (see section 4). This effect is calledinfusion-related reactionand, in some cases, can be severe.

• Infusion-related reactions include dizziness, headache, nausea, low blood pressure, fatigue, and fever. If you experience an infusion-related reaction,you must inform your doctor immediately.
• If you have an infusion-related reaction, you may be given other medications to treat it or to help prevent future reactions. These medications may include medications used to treat allergies (antihistamines), medications used to treat fever (antipyretics), and medications to control inflammation (corticosteroids).
• If the infusion-related reaction is severe, your doctor will immediately stop the infusion and begin administering appropriate medical treatment or slow down the treatment.
• If infusion-related reactions are severe or there is a loss of effect of this medication, your doctor will perform a blood test to check if you have antibodies that may affect the treatment outcome.
• Most of the time, you can continue receiving Elfabrio even if you experience an infusion-related reaction.

In very rare cases, your immune system may not be able to recognize Elfabrio and cause a kidney immune disease (membranoproliferative glomerulonephritis). During clinical studies, only one case occurred, and the only symptoms reported were a temporary decrease in kidney function with excess protein in the urine. Symptoms resolved after treatment was stopped.

Children and adolescents

This medication should not be used in children and adolescents. The safety and efficacy of Elfabrio have not been established in children and adolescents from 0 to 17 years.

Other medications and Elfabrio

Inform your doctor if you are taking, have taken recently, or may need to take any other medication.

Pregnancy and breastfeeding

You should not receive Elfabrio if you are pregnant, as there is no experience with Elfabrio in pregnant women. If you are pregnant, think you may be pregnant, or intend to become pregnant, consult your doctor before receiving this medication.

The excretion of Elfabrio in breast milk is unknown. Inform your doctor if you are breastfeeding or intend to do so. Your doctor will help you decide if you need to stop breastfeeding or stop Elfabrio treatment after considering the benefits of breastfeeding for the baby and the benefits of Elfabrio for you.

Driving and operating machines

Elfabrio may cause dizziness or vertigo. If you feel dizzy or have vertigo on the day of Elfabrio treatment, do not drive or use machines until you feel better.

Elfabrio contains sodium

This medication contains 46mg of sodium (main component of table salt/for cooking) in each 10ml vial. This is equivalent to 2% of the maximum daily sodium intake recommended for an adult.

This medication contains 11.5mg of sodium (main component of table salt/for cooking) in each 2.5ml vial. This is equivalent to 1% of the maximum daily sodium intake recommended for an adult.

This medication should only be used under the supervision of a doctor with experience in treating Fabry disease or similar conditions and should only be administered by a healthcare professional.

The recommended dose is 1 mg/kg of body weight administered once every two weeks.

Your doctor may instruct you to be treated at home if you meet certain criteria. Contact your doctor if you wish to be treated at home.

See the information for healthcare professionals at the end of this leaflet.

Like all medicines, this medicine can cause side effects, although not everyone will experience them. Most side effects occur during infusion or shortly after it ("infusion-related reaction," see section 2 "Warnings and Precautions").

While you are being treated with Elfabrio, you may experience some of the following reactions:

Severe side effects

Frequent side effects (may affect up to 1 in 10 people)

• severe hypersensitivity and anaphylactic reaction (symptoms include prolonged and excessive contraction of the respiratory tract muscles causing difficulty breathing [bronchospasm]), facial, mouth, and throat swelling, wheezing [“popping” while breathing], low blood pressure, urticaria, difficulty swallowing, skin rash, shortness of breath, flushing, chest discomfort, itching, sneezing, and nasal congestion)

If these side effects occur, seek immediate medical attention and discontinue infusion. Your doctor will administer medical treatment as needed.

Other side effects are

Frequent (may affect up to 1 in 10 people)

• infusion-related reactions
• weakness
• nausea
• skin rash
• abdominal pain
• dizziness
• pain
• chest pain
• headache
• muscle and joint pain
• sensations such as numbness, tingling, or prickling (paresthesia)
• itching (pruritus)
• diarrhea
• vomiting
• chills
• skin redness (erythema)
• sensation of spinning (vertigo), activation, irritability, or confusion
• alteration of normal heart rhythm
• agitation

Rare (may affect up to 1 in 100 people)

• tremor
• high blood pressure (hypertension)
• bronchospasm (contraction of bronchial muscles causing obstruction of respiratory passages) and difficulty breathing
• throat irritation
• elevated body temperature
• difficulty sleeping (insomnia)
• restless legs syndrome
• nerve damage in arms and legs causing pain or numbness, burning, and tingling (peripheral neuropathy)
• nerve pain (neuralgia)
• sensation of burning
• flushing
• gastroesophageal reflux disease (GERD) in which stomach acid rises into the esophagus
• inflammation of the stomach lining (mucosa) (dyspepsia)
• indigestion
• gas (flatulence)
• decreased sweating (hypohidrosis)
• immunological renal disease causing excess protein in urine and renal dysfunction (membranoproliferative glomerulonephritis)
• chronic nephropathy
• excess protein in urine (proteinuria)
• tissue damage due to the medication that normally infuses into a vein leaking or being accidentally infused into surrounding tissue (extravasation at the infusion site)
• swelling in the lower legs or hands (edema)
• swelling in the arms or legs
• influenza-like illness
• nasal congestion and sneezing
• pain at the infusion site
• elevation of liver enzymes and uric acid in the blood, elevation of the protein/creatinine ratio in urine, and presence of leukocytes in urine in laboratory tests
• weight gain
• low blood pressure (hypotension)
• low heart rate (bradycardia)
• thickening of the inner wall of the heart ventricle

Reporting side effects

If you experience any type of side effect, consult your doctor, even if it is a possible side effect that does not appear in this prospectus. You can also report them directly through thenational notification system included in theSpanish System for Pharmacovigilance of Medicines for Human Use https://notificaram.es. By reporting side effects, you can contribute to providing more information on the safety of this medicine.

Keep this medication out of the sight and reach of children.

Do not use this medication after the expiration date that appears on the label and on the box after «CAD». The expiration date is the last day of the month indicated.

Store in refrigerator (between 2°C and 8°C).

After dilution, the diluted solution must be used immediately. If not used immediately, the diluted solution must be stored for a maximum of 24hours in refrigerator (between 2°C and 8°C) or for a maximum of 8 hours at room temperature (below 25°C).

Do not use this medication if you observe solid particles or a change in color.

Medications should not be disposed of through drains or in the trash. Ask your pharmacist how to dispose of the containers and medications that you no longer need. This will help protect the environment.

Composition of Elfabrio

• The active principle is pegunigalsidase alfa. Each vial contains 20 mg of pegunigalsidase alfa in 10 ml or 5 mg of pegunigalsidase alfa in 2.5 ml (2 mg/ml).
• The other components are: tribasic dihydrogen sodium citrate, citric acid, and sodium chloride (see section 2 "Elfabrio contains sodium").

Appearance of the product and contents of the container

Transparent and colourless solution in a transparent glass vial with a rubber stopper and sealed with an aluminium closure.

Container sizes: 1, 5 or 10 vials.

Only some container sizes may be marketed.

Marketing authorisation holder

Chiesi Farmaceutici S.p.A.

Via Palermo 26/A

43122 Parma

Italy

Responsible person

Chiesi Farmaceutici S.p.A.

Via San Leonardo 96

43122 Parma

Italy

For further information about this medicinal product, please consult the representative of the marketing authorisation holder in your country:

Last update of this leaflet: May 2024

Other sources of information

The detailed information on this medicinal product is available on the website of the European Medicines Agency:http://www.ema.europa.eu.There are also links to other websites on rare diseases and orphan medicinal products.

--------------------------------------------------------------------------------------------------------------

This information is intended for healthcare professionals only:

Dilution (using an aseptic technique)

1. Determine the total number of vials required for the infusion.

The number of vials required is based on the total dose required for each patient and requires calculating a dose based on weight.

An example of calculating the total dose in a 80 kg patient who has been prescribed a dose of 1 mg/kg is as follows:

• Patient weight (in kg) ÷ 2 = dose volume (in ml)
• Example: 80 kg (patient weight) ÷ 2 = 40 ml (volume that must be withdrawn).
• In this example, 4 vials of the 10 ml vial (or 16 vials of the 2.5 ml vial) are required.

1. Allow the required number of vials to reach room temperature before dilution (approximately 30 minutes).

Visually inspect the vials. Do not use if the closure cap is missing or broken. Do not use if there are solid particles or if there has been a colour change.

Avoid shaking or agitating the vials.

1. Withdraw and discard the same volume calculated in step 1 from a 0.9% sodium chloride infusion solution bag.

1. Withdraw the required volume from the Elfabrio solution vials and dilute with a 0.9% sodium chloride infusion solution to the total volume based on the patient's weight as specified in the table below.

Minimum infusion volume for patients by body weight

Inject the Elfabrio solution directly into the infusion bag.

DO NOT inject into the air space within the infusion bag.

Gently invert the infusion bag to mix the solution, avoiding vigorous shaking or agitation.

The diluted solution must be administered using a 0.2 μm in-line filter with low protein binding.