Refixia

nonacog beta pegol

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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.

What is Refixia and what is it used for?

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.

How is Refixia used?

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.

How does Refixia work?

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.

What benefits of Refixia have been shown in studies?

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.

What are the risks associated with Refixia?

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.

Why is Refixia approved?

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.

What measures are being taken to ensure the safe and effective use of Refixia?

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.

Other information about Refixia

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.

Name Language First published Last updated
Refixia : EPAR - Summary for the public BG = bălgarski 2017-08-02  
Refixia : EPAR - Summary for the public ES = español 2017-08-02  
Refixia : EPAR - Summary for the public CS = čeština 2017-08-02  
Refixia : EPAR - Summary for the public DA = dansk 2017-08-02  
Refixia : EPAR - Summary for the public DE = Deutsch 2017-08-02  
Refixia : EPAR - Summary for the public ET = eesti keel 2017-08-02  
Refixia : EPAR - Summary for the public EL = elliniká 2017-08-02  
Refixia : EPAR - Summary for the public EN = English 2017-08-02  
Refixia : EPAR - Summary for the public FR = français 2017-08-02  
Refixia : EPAR - Summary for the public IT = italiano 2017-08-02  
Refixia : EPAR - Summary for the public LV = latviešu valoda 2017-08-02  
Refixia : EPAR - Summary for the public LT = lietuvių kalba 2017-08-02  
Refixia : EPAR - Summary for the public HU = magyar 2017-08-02  
Refixia : EPAR - Summary for the public MT = Malti 2017-08-02  
Refixia : EPAR - Summary for the public NL = Nederlands 2017-08-02  
Refixia : EPAR - Summary for the public PL = polski 2017-08-02  
Refixia : EPAR - Summary for the public PT = português 2017-08-02  
Refixia : EPAR - Summary for the public RO = română 2017-08-02  
Refixia : EPAR - Summary for the public SK = slovenčina 2017-08-02  
Refixia : EPAR - Summary for the public SL = slovenščina 2017-08-02  
Refixia : EPAR - Summary for the public FI = suomi 2017-08-02  
Refixia : EPAR - Summary for the public SV = svenska 2017-08-02  
Refixia : EPAR - Summary for the public HR = Hrvatski 2017-08-02  

This EPAR was last updated on 02/08/2017 .

Authorisation details

Product details

Product details for Refixia
NameRefixia
Agency product numberEMEA/H/C/004178
Active substance

nonacog beta pegol

International non-proprietary name (INN) or common name

nonacog beta pegol

Therapeutic area Hemophilia B
Anatomical therapeutic chemical (ATC) code B02BD04
Additional monitoring

This medicine is under additional monitoring. This means that it is being monitored even more intensively than other medicines. For more information, see medicines under additional monitoring.

Publication details

Publication details for Refixia
Marketing-authorisation holder

Novo Nordisk A/S

Revision0
Date of issue of marketing authorisation valid throughout the European Union02/06/2017

Contact address:

Novo Nordisk A/S
Novo Alle 2880
Bagsvaerd
Denmark

Product information

Product information

02/06/2017  Refixia -EMEA/H/C/004178 --

Name Language First published Last updated
Refixia : EPAR - Product Information HR = Hrvatski 2017-08-02  
Refixia : EPAR - Product Information HR = Hrvatski 2017-08-02  
Refixia : EPAR - Product Information HR = Hrvatski 2017-08-02  
Refixia : EPAR - Product Information HR = Hrvatski 2017-08-02  
Refixia : EPAR - Product Information HR = Hrvatski 2017-08-02  
Refixia : EPAR - Product Information HR = Hrvatski 2017-08-02  
Refixia : EPAR - Product Information HR = Hrvatski 2017-08-02  
Refixia : EPAR - Product Information HR = Hrvatski 2017-08-02  
Refixia : EPAR - Product Information HR = Hrvatski 2017-08-02  
Refixia : EPAR - Product Information HR = Hrvatski 2017-08-02  
Refixia : EPAR - Product Information HR = Hrvatski 2017-08-02  
Refixia : EPAR - Product Information HR = Hrvatski 2017-08-02  
Refixia : EPAR - Product Information HR = Hrvatski 2017-08-02  
Refixia : EPAR - Product Information HR = Hrvatski 2017-08-02  
Refixia : EPAR - Product Information HR = Hrvatski 2017-08-02  
Refixia : EPAR - Product Information HR = Hrvatski 2017-08-02  
Refixia : EPAR - Product Information HR = Hrvatski 2017-08-02  
Refixia : EPAR - Product Information HR = Hrvatski 2017-08-02  
Refixia : EPAR - Product Information HR = Hrvatski 2017-08-02  
Refixia : EPAR - Product Information HR = Hrvatski 2017-08-02  
Refixia : EPAR - Product Information HR = Hrvatski 2017-08-02  
Refixia : EPAR - Product Information HR = Hrvatski 2017-08-02  
Refixia : EPAR - Product Information HR = Hrvatski 2017-08-02  
Refixia : EPAR - Product Information HR = Hrvatski 2017-08-02  
Refixia : EPAR - Product Information HR = Hrvatski 2017-08-02  

Contents

  • Annex I - Summary of product characteristics
  • Annex IIA - Manufacturing-authorisation holder responsible for batch release
  • Annex IIB - Conditions of the marketing authorisation
  • Annex IIIA - Labelling
  • Annex IIIB - Package leaflet

Please note that the size of the above document can exceed 50 pages.

You are therefore advised to be selective about which sections or pages you wish to print.


Name Language First published Last updated
Refixia : EPAR - All Authorised presentations HR = Hrvatski 2017-08-02  
Refixia : EPAR - All Authorised presentations HR = Hrvatski 2017-08-02  
Refixia : EPAR - All Authorised presentations HR = Hrvatski 2017-08-02  
Refixia : EPAR - All Authorised presentations HR = Hrvatski 2017-08-02  
Refixia : EPAR - All Authorised presentations HR = Hrvatski 2017-08-02  
Refixia : EPAR - All Authorised presentations HR = Hrvatski 2017-08-02  
Refixia : EPAR - All Authorised presentations HR = Hrvatski 2017-08-02  
Refixia : EPAR - All Authorised presentations HR = Hrvatski 2017-08-02  
Refixia : EPAR - All Authorised presentations HR = Hrvatski 2017-08-02  
Refixia : EPAR - All Authorised presentations HR = Hrvatski 2017-08-02  
Refixia : EPAR - All Authorised presentations HR = Hrvatski 2017-08-02  
Refixia : EPAR - All Authorised presentations HR = Hrvatski 2017-08-02  
Refixia : EPAR - All Authorised presentations HR = Hrvatski 2017-08-02  
Refixia : EPAR - All Authorised presentations HR = Hrvatski 2017-08-02  
Refixia : EPAR - All Authorised presentations HR = Hrvatski 2017-08-02  
Refixia : EPAR - All Authorised presentations HR = Hrvatski 2017-08-02  
Refixia : EPAR - All Authorised presentations HR = Hrvatski 2017-08-02  
Refixia : EPAR - All Authorised presentations HR = Hrvatski 2017-08-02  
Refixia : EPAR - All Authorised presentations HR = Hrvatski 2017-08-02  
Refixia : EPAR - All Authorised presentations HR = Hrvatski 2017-08-02  
Refixia : EPAR - All Authorised presentations HR = Hrvatski 2017-08-02  
Refixia : EPAR - All Authorised presentations HR = Hrvatski 2017-08-02  
Refixia : EPAR - All Authorised presentations HR = Hrvatski 2017-08-02  
Refixia : EPAR - All Authorised presentations HR = Hrvatski 2017-08-02  
Refixia : EPAR - All Authorised presentations HR = Hrvatski 2017-08-02  

Pharmacotherapeutic group

Antihemorrhagics

Therapeutic indication

Treatment and prophylaxis of bleeding in patients 12 years and above with haemophilia B (congenital factor IX deficiency).

Assessment History

Changes since initial authorisation of medicine

Name Language First published Last updated

Initial marketing-authorisation documents

Name Language First published Last updated
Refixia : EPAR - Public assessment report HR = Hrvatski 2017-08-02  
CHMP summary of positive opinion for Refixia HR = Hrvatski 2017-03-24  

Authorised

This medicine is approved for use in the European Union

More information on Refixia

This product is no longer an orphan medicine. It was originally designated an orphan medicine on 15 May 2009. Refixia was withdrawn from the Community register of orphan medicinal products in May 2017 upon request of the marketing authorisation holder at the time of the granting of a marketing authorisation.