Please note that this product was withdrawn from the Union Register of orphan medicinal products in October 2024 on request of the Sponsor.
On 19 November 2014, orphan designation (EU/3/14/1360) was granted by the European Commission to Apitope International NV, Belgium, for a combination of H-Lys-Lys-Gly-Pro-Arg-Cys(SH)-Leu-Thr-Arg-Tyr-Tyr-Ser-Ser-Phe-Val-Asn-Met-Glu-Gly-Lys-Lys-OH and H-Lys-Lys-Gly-Asp-Asn-Ile-Met-Val-Thr-Phe-Arg-Asn-Gln-Ala-Ser-Arg-Pro-Tyr-Gly-Lys-Lys-OH 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 was equivalent to a total of around 36,000 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).
*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 511,100,000 (Eurostat 2014).
At the time of designation, medicines containing factor VIII were authorised in the EU for the treatment of haemophilia A, to replace the missing protein. However, not all patients with haemophilia A could benefit from these medicines because the immune system (the body's natural defences) can react against them by producing 'inhibitors' (antibodies) against factor VIII. 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 A because studies in experimental models suggest that it works in a different way to existing treatments by suppressing the production of antibodies against factor VIII. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
This medicine is made up of two small protein fragments based on parts of factor VIII. Although it is not fully clear how the medicine works, it is thought to activate certain cells of the immune system (known as suppressor T cells) that suppress the production of antibodies against factor VIII. This would allow patients with haemophilia A to benefit from treatment with medicines containing factor VIII.
he effects of this 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 A had been started.
At the time of submission, the 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 9 October 2014 recommending the granting of this designation.
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.
S-Cubed Pharmaceutical Services ApS
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
| Date | Update |
|---|---|
| November 2022 | The sponsorship was transferred from Apitope International N.V., Belgium to S-Cubed Pharmaceutical Services ApS, Denmark. |
| June 2023 | The sponsor’s address was updated in June 2023. |
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
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.
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: