EU/3/18/2067 - orphan designation for treatment in haematopoietic stem cell transplantation
recombinant human monoclonal antibody against mannan-binding lectin-associated serine protease-2
OrphanHuman
Overview
On 24 August 2018, orphan designation (EU/3/18/2067) was granted by the European Commission to Omeros London Limited, United Kingdom, for recombinant human monoclonal antibody against mannan-binding lectin-associated serine protease-2 (also known as OMS721) for treatment in haematopoietic stem cell transplantation.
The sponsorship was transferred to Omeros Ireland Limited, Ireland, in January 2021.
Haematopoietic stem cell transplantation (HSCT) is a procedure where the patient's bone marrow is cleared of cells and replaced by stem cells (cells that can develop into different types of cell) from a donor to form new bone marrow that produces healthy blood cells. It can be used to treat serious diseases of the blood and immune system such as leukaemia.
HSCT can be a debilitating and life-threatening procedure due to the risk of severe infections and developing graft-versus-host disease (when the transplanted cells regard the patient’s body as ‘foreign’ and attack the patient’s organs, leading to organ damage). It can also lead to a condition known as thrombotic microangiopathy, where blood clots occur in small blood vessels.
At the time of designation, approximately 1 in 10,000 people in the European Union (EU) receive HSCT per year in the European Union (EU). This was equivalent to a total of around 52,000 people*, and is below the ceiling for orphan designation. 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 517,400,000 (Eurostat 2018).
At the time of designation, several medicines were authorised in the EU for patients undergoing HSCT. These included radiation treatment or intensive treatment with cancer medicines such as busulfan to clear the bone marrow of existing cells, medicines to help restore the immune system, such as filgrastim, immunoglobulin replacement therapy and Zalmoxis, and medicines to reduce the risk of infections, such as antiviral and antifungal medicines. Medicines that suppress the immune system, such as ciclosporin and corticosteroids, were used for the treatment of graft-versus-host disease. However, there were no products authorised for thrombotic microangiopathy.
The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients undergoing HSCT. Studies showed improved survival compared with other existing treatments when this medicine was used to treat patients with thrombotic microangiopathy after HSCT.
This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
The medicine is a monoclonal antibody designed to attach to a protein (MASP-2) involved in the formation of blood clots seen in thrombotic microangiopathy. Attaching to and blocking the action of this protein is expected to prevent blood clots in small blood vessels and thereby improve outcomes in patients undergoing HSCT.
The effects of the medicine have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with the medicine in patients undergoing HSCT were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for in patients undergoing HSCT. Orphan designation of this medicine had been granted in the United States for complement-mediated thrombotic microangiopathy.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 19 July 2018 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
- recombinant human monoclonal antibody against mannan-binding lectin-associated serine protease-2
- Intended use
- Treatment in haematopoietic stem cell transplantation
- Orphan designation status
- Positive
- EU designation number
- EU/3/18/2067
- Date of designation
- Sponsor
Ormond Building
31-36 Ormond Quay Upper
Dublin 7
D07 EE37
Co. Dublin
Ireland
Email: info@omeros.ie
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
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: