This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information.
The text only version may be available in large print, Braille or audio CD. For further information call emc accessibility on 0800 198 5000. The product code(s) for this leaflet is: PLGB 08829/0210.
Elfabrio 2 mg/mL concentrate for solution for infusion
Elfabrio 2 mg/mL
concentrate for solution for infusion
▼This medicine is subject to additional monitoring. This will allow quick identification of new safety information. You can help by reporting any side effects you may get. See the end of section 4 for how to report side effects.
1. What Elfabrio is and what it is used for
2. What you need to know before you are given Elfabrio
3. How Elfabrio is given
4. Possible side effects
5. How to store Elfabrio
6. Contents of the pack and other information
Elfabrio contains the active substance pegunigalsidase alfa, and is used as enzyme replacement therapy in adult patients with confirmed Fabry disease. Fabry disease is a rare genetic disease that can affect many parts of the body. In patients with Fabry disease, a fat substance is not removed from the cells of their body, and builds up in the walls of blood vessels which can cause organ failure. This fat builds up in the cells of these patients because they do not have enough 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 severely allergic to pegunigalsidase alfa or any of the other ingredients of this medicine (listed in section 6).
Talk to your doctor before Elfabrio is used.
If you are treated with Elfabrio, you may experience a side effect during or immediately following the drip (infusion) used to give the medicine (see section 4). This is known as an infusion-related reaction and can sometimes be severe.
In very rare cases, your immune system may not be able to recognise Elfabrio, leading to an immunological kidney disease (glomerulonephritis membranoproliferative). During the clinical studies, only one case occurred, and the only symptoms reported were a temporary decline of renal functions with excess proteins in the urine. The symptoms resolved upon discontinuation of the treatment.
This medicine should not be used in children and adolescents. The safety and efficacy of Elfabrio in children and adolescents aged 0-17 years have not been established.
Tell your doctor if you are taking, have recently taken or might take any other medicines.
You should not use Elfabrio if you are pregnant, since there is no experience with Elfabrio in pregnant women. If you are pregnant, think you may be pregnant or are planning to have a baby, ask your doctor for advice before you are given this medicine.
It is unknown whether Elfabrio is excreted in human milk. Tell your doctor if you are breast-feeding or plan to do so. Your doctor will then help you decide whether to stop breast-feeding or stop taking Elfabrio, considering the benefit of breast-feeding for your baby and the benefit of Elfabrio for you.
Elfabrio may cause dizziness or vertigo. If you feel dizzy or vertigo on the day of treatment with Elfabrio, do not to drive or use machines until you feel better.
This medicine contains 48 mg sodium (main component of cooking/table salt) in each vial. This is equivalent to 2% of the recommended maximum daily dietary intake of sodium for an adult.
This medicine is only to be used under the supervision of a doctor experienced in the treatment of Fabry disease or other similar diseases and should only be given by a healthcare professional.
The recommended dose is 1 mg/kg of body weight given once every two weeks.
Your doctor may advise that you can be treated at home provided you meet certain criteria.
Please contact your doctor if you would like to be treated at home.
See information for healthcare professionals at the end of this package leaflet.
Like all medicines, this medicine can cause side effects, although not everybody gets them. Most side effects occur during the infusion or shortly after (“infusion-related reaction”, see section 2 “Warnings and precautions”).
While under treatment with Elfabrio, you may experience some of the following reactions:
Common side effects (may affect up to 1 in 10 people)
If these side effects happen, immediately seek medical attention and stop the infusion. Your doctor will give you medical treatment if required.
Common (may affect up to 1 in 10 people)
Uncommon (may affect up to 1 in 100 people)
If you get any side effects, talk to your doctor. This includes any possible side effects not listed in this leaflet. You can also report side effects directly via:
or search for MHRA Yellow Card in the Google Play or Apple App Store
By reporting side effects you can help provide more information on the safety of this medicine.
Keep this medicine out of the sight and reach of children.
Do not use this medicine after the expiry date which is stated on the label and carton after “EXP”.
The expiry date refers to the last day of that month.
Store in a refrigerator (2 °C-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-8 °C) or for no longer than 8 hours at room temperature (below 25 °C).
Do not use this medicine if you notice particles or discolouration.
Do not throw away any medicines via wastewater or household waste. Ask your pharmacist how to throw away medicines you no longer use. These measures will help protect the environment.
Clear and colourless solution in clear glass vial with a rubber stopper and sealed with aluminium flip off cap.
Pack sizes: 1, 5 or 10 vials.
Not all pack sizes may be marketed.
United Kingdom (Northern Ireland) & Ireland:
For any information about this medicine, please contact the local representative of the Marketing Authorisation Holder:
This leaflet was last revised in May 2023
Other sources of information
Detailed information on this medicine is available on the European Medicines Agency web site: http://www.ema.europa.eu. There are also links to other websites about rare diseases and treatments.