EU/3/12/1052 - orphan designation for treatment of haemophilia A
Humanised monoclonal IgG4 antibody against tissue-factor-pathway inhibitor (concizumab)
OrphanHuman
Overview
Please note that this product was withdrawn from the Union Register of orphan medicinal products in December 2022 on request of the Sponsor.
On 10 October 2012, orphan designation (EU/3/12/1052) was granted by the European Commission to Novo Nordisk A/S, Denmark, for humanised monoclonal IgG4 antibody against tissue-factor-pathway inhibitor for the treatment of haemophilia A.
Haemophilia A is an inherited bleeding disorder that is caused by the lack of factor VIII, which is one of the proteins involved in the blood coagulation (clotting) process. Patients with haemophilia A are more prone to bleeding than normal and have prolonged bleeding after injury or surgery. Bleeding can also happen within muscles or the spaces in the joints, such as the elbows, knees and ankles. This can lead to permanent injury if it happens repeatedly.
Haemophilia A is a debilitating disease that is life-long and may be life threatening because bleeding can happen in the brain, the spinal cord or the gut.
At the time of designation, haemophilia A affected approximately 0.7 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 35,000 people, and is below the ceiling for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).
At the time of submission of the application for orphan drug designation, medicines containing factor VIII were authorised in the EU for the treatment of haemophilia A, to replace the missing protein. Some patients needed other treatments, such as medicines containing other substances involved in blood clotting.
The sponsor has provided sufficient information to show that the medicine 'humanised monoclonal IgG4 antibody against tissue-factor-pathway inhibitor' might be of significant benefit for patients with haemophilia A because it works in a different way to existing treatments, and early studies in experimental models show that it might prevent bleeding in patients with this condition. In addition, the medicine will also be available as a solution for subcutaneous (under the skin) injection, which will be more convenient to use than currently authorised treatments, which are given as injections into a vein. These assumptions will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
This medicine is a monoclonal antibody (a type of protein) that has been designed to recognise and attach to a molecule in the body called 'tissue-factor-pathway inhibitor' (TFPI). TFPI blocks the production of factor Xa, a protein central to the clotting process. The production of factor Xa is controlled by two separate pathways, one involving factor VIII, the other involving TFPI. By attaching to TFPI, this medicine is expected to block its action, resulting in a prolonged production of factor Xa. This is expected to increase the ability of the blood to clot and help control the bleeding disorder, bypassing the need for factor VIII.
The effects of humanised monoclonal IgG4 antibody against tissue-factor-pathway inhibitor have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with the medicine including patients with haemophilia A were ongoing.
At the time of submission, this medicine was not authorised anywhere in the EU for haemophilia A or designated as an orphan medicinal product elsewhere for this condition.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 12 September 2012 recommending the granting of this designation.
- the seriousness of the condition;
- the existence of alternative methods of diagnosis, prevention or treatment;
- either the rarity of the condition (affecting not more than 5 in 10,000 people in the EU) or insufficient returns on investment.
Designated orphan medicinal products are products that are still under investigation and are considered for orphan designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.
Key facts
- Active substance
- Humanised monoclonal IgG4 antibody against tissue-factor-pathway inhibitor (concizumab)
- Intended use
- Treatment of haemophilia A
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/12/1052
- Date of designation
- Sponsor
Novo Nordisk A/S
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
Update history
| Date | Update |
|---|---|
| December 2022 | The product was withdrawn from the Union Register of orphan medicinal products on request of the Sponsor. |
EMA list of opinions on orphan medicinal product designation
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
Patients' organisations
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: