Overview
This medicine is now known as dalcinonacog alfa.
On 20 June 2017, orphan designation (EU/3/17/1884) was granted by the European Commission to Voisin Consulting S.A.R.L., France, for recombinant human factor IX protein modified with three point mutations (also known as CB 2679d or ISU304) for the treatment of haemophilia B.
Haemophilia B is an inherited bleeding disorder that is caused by the lack of factor IX, which is one of the proteins involved in the blood coagulation (clotting) process. Patients with haemophilia B are more prone to bleeding than normal and have poor wound healing 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 B is a debilitating disease that is life long and may be life threatening because bleeding can also happen in the brain and spinal cord, the throat or the gut.
At the time of designation, haemophilia B affected approximately 0.25 in 10,000 people in the European Union (EU). This was equivalent to a total of around 13,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).
*Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed on the basis of data from the European Union (EU 28), Norway, Iceland and Liechtenstein. This represents a population of 515,700,000 (Eurostat 2017).
At the time of designation, medicines containing factor IX were authorised in the EU for the treatment of haemophilia B, to replace the missing protein. However, factor IX medicines did not work in some patients with haemophilia B because the immune system (the body's natural defences) can produce 'inhibitors' (antibodies) against factor IX which stop the factor IX medicine from working. In these cases, other treatments needed to be used, such as factor VIIa (the activated form of factor VII, another protein involved in blood clotting), either alone or as part of a combination treatment.
The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with haemophilia B. Data from laboratory studies showed that the medicine, which is given daily by injection under the skin, can improve clotting to a similar degree as current treatments which have to be given by injection into a vein, and that this represents a major contribution to patient care. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
This medicine contains a version of factor IX, the protein lacking in patients with haemophilia B. This protein has been engineered to enhance its clotting activity and to increase the length of time it remains in the body. After being given to the patient as an injection under the skin, the factor IX is expected to pass into the blood where it can replace the missing protein and so control the bleeding disorder.
The effects of the medicine have been evaluated in experimental models.
At the time of submission of the application for orphan designation, no clinical trials with the medicine in patients with haemophilia B had been started.
At the time of submission, the medicine was not authorised anywhere in the EU for haemophilia B 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 May 2017 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.
EU/3/17/1884: Public summary of opinion on orphan designation: Recombinant human factor IX protein modified with three point mutations for the treatment of haemophilia B
Key facts
- Active substance
- Recombinant human factor IX protein modified with three point mutations (dalcinonacog alfa)
- Intended use
- Treatment of haemophilia B
- Orphan designation status
- Positive
- EU designation number
- EU/3/17/1884
- Date of designation
- Sponsor
Premier Research Group S.L.
Camino Zarzuela 19
28023 Madrid
Spain
E-mail: regulatory@gccorp.com
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 |
---|---|
August 2023 | The sponsorship was transferred to Premier Research Group S.L., Spain in August 2023. |
July 2020 | The sponsorship was transferred to Turnkey PharmaConsulting Ireland Limited, Ireland. |
October 2019 | The sponsorship was transferred to Regintel Limited, Ireland. |
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:
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: