Refixia
Authorised
This medicine is authorised for use in the European Union
nonacog beta pegol
MedicineHumanAuthorised
- Application under evaluation
- CHMP opinion
- European Commission decision
Overview
This is a summary of the European public assessment report (EPAR) for Refixia. It explains how the Agency assessed the medicine to recommend its authorisation in the EU and its conditions of use. It is not intended to provide practical advice on how to use Refixia.
For practical information about using Refixia, patients should read the package leaflet or contact their doctor or pharmacist.
Refixia is a medicine used to treat and prevent bleeding in patients with haemophilia B, an inherited bleeding disorder caused by lack of a clotting protein called factor IX. It can be used in adults and children from 12 years of age.
Refixia contains the active substance nonacog beta pegol.
Refixia can only be obtained with a prescription and treatment should be under the supervision of a doctor who has experience in the treatment of haemophilia.
Refixia is available as a powder and a liquid that are mixed together to make a solution for injection into a vein. The dose and frequency of treatment depend on whether Refixia is used to treat or prevent bleeding, or to reduce bleeding during surgery, the extent and location of the bleeding, and the patient’s bodyweight. For further information on how to use this medicine, see the summary of product characteristics (also part of the EPAR).
Patients or their carers may be able to inject Refixia themselves at home once they have been trained appropriately. For full details, see the package leaflet.
Patients with haemophilia B lack factor IX, a protein needed for normal clotting of the blood, and as a result, they bleed readily. The active substance in Refixia, nonacog beta pegol, works in the body in the same way as human factor IX. It replaces the missing factor IX, thereby helping the blood to clot and giving temporary control of bleeding.
Refixia has been shown to be effective at both treating bleeding episodes and keeping the number of episodes low.
In a study involving 74 adults and adolescents aged 13 or above, 29 patients given Refixia as a weekly preventive treatment had around 1 bleeding episode a year, and 15 patients given Refixia for treating bleedings ‘on demand’ had around 16 bleeding episodes a year. In addition, when bleeding did occur, Refixia was rated excellent or good at treating around 92% of bleeding episodes. 87% of bleeding episodes resolved with one injection of Refixia.
In the second study in 25 children aged below 13 years, all patients were given Refixia as a weekly preventive treatment. Patients had around 1 bleeding episode per year and Refixia was rated excellent or good at treating around 93% of bleeding episodes. Around 86% of bleeding episodes resolved with one injection.
Hypersensitivity (allergic) reactions are uncommon with Refixia (may affect up to 1 in 100 patients) and may include: swelling, burning and stinging at the injection site, chills, flushing, itchy rash, headache, hives, low blood pressure, lethargy, nausea and vomiting, restlessness, a fast heartbeat, tightness of the chest and wheezing. In some cases these reactions can become severe.
Some patients taking factor IX medicines may develop inhibitors (antibodies) against factor IX, causing the medicine to stop working and resulting in a loss of bleeding control. Factor IX medicines can also potentially cause problems due to the formation of blood clots in the blood vessels.
Refixia must not be used in patients allergic to hamster proteins. For the full list of side effects and restrictions, see the package leaflet.
The Agency’s Committee for Medicinal Products for Human Use (CHMP) decided that Refixia’s benefits are greater than its risks and recommended that it be approved for use in the EU.
Studies show that Refixia is effective at preventing and treating bleeding episodes in patients with haemophilia B and its safety is comparable to that of other factor IX products. However, part of the active substance in Refixia (called PEG) may accumulate in the body, including in a structure in the brain called choroid plexus, following long-term treatment. Since this could potentially cause problems especially in children below 12 years of age, Refixia is only approved for use in in adults and children from 12 years of age.
The company that markets Refixia will conduct a study to investigate the potential effects of PEG accumulation in the choroid plexus of the brain and other organs.
Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Refixia have been included in the summary of product characteristics and the package leaflet.
The European Commission granted a marketing authorisation valid throughout the European Union for Refixia on 02 June 2017.
For more information about treatment with Refixia, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.
Product information
Latest procedure affecting product information:
II/0032
04/08/2023
This medicine’s product information is available in all official EU languages.
Select 'available languages' to access the language you need.
Product information documents contain:
- summary of product characteristics (annex I);
- manufacturing authorisation holder responsible for batch release (annex IIA);
- conditions of the marketing authorisation (annex IIB);
- labelling (annex IIIA);
- package leaflet (annex IIIB).
Product details
- Name of medicine
- Refixia
- Active substance
- Nonacog beta pegol
- International non-proprietary name (INN) or common name
- nonacog beta pegol
- Therapeutic area (MeSH)
- Hemophilia B
- Anatomical therapeutic chemical (ATC) code
- B02BD04
Pharmacotherapeutic group
AntihemorrhagicsTherapeutic indication
Treatment and prophylaxis of bleeding in patients with haemophilia B (congenital factor IX deficiency).
Refixia can be used for all age groups.
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