EU/3/16/1609 - orphan designation for treatment of autoimmune haemolytic anaemia
sutimlimab
OrphanHuman
Overview
On 17 February 2016, orphan designation (EU/3/16/1609) was granted by the European Commission to Assign Group Development UK Ltd, United Kingdom, for humanised IgG4 monoclonal antibody against total complement component 1, subcomponent s for the treatment of autoimmune haemolytic anaemia.
This medicine is now known as sutimlimab.
The medicinal product has been authorised in the EU as Enjaymo since 15 November 2022.
Autoimmune haemolytic anaemia is a condition that occurs when the body recognises red blood cells as foreign and attacks them, eventually destroying them. This leads to low red blood cell counts and low levels of haemoglobin, which in turn causes symptoms such as tiredness and inability to exercise.
Autoimmune haemolytic anaemia is a long-term debilitating condition which may cause blood clots in the veins or arteries and infections, and may require transfusions of red blood cells.
At the time of designation, autoimmune haemolytic anaemia affected less than 2.3 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 118,000 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 513,700,000 (Eurostat 2016).
At the time of designation, corticosteroids (medicines that suppress the activity of the immune system) were authorised in the EU for the treatment of autoimmune haemolytic anaemia. Red blood cell transfusion and splenectomy (removing the spleen) was also performed in some patients.
The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with autoimmune haemolytic anaemia. This is because early studies in patients show that the medicine prevents breaking of the red blood cells and improves haemoglobin levels, which may have benefits in the short and long term when compared with existing methods. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
This medicine has been designed to attach to a protein called 'complement component 1, subcomponent s' (C1s), which is part of the body's defence system called 'complement'. In autoimmune haemolytic anaemia, patients' red blood cells often have proteins on their surface that allow complement to attack the cells. This results in complement destroying the red blood cells. By blocking C1s the medicine is expected to prevent the complement system from attacking the cells, thus reducing their destruction and improving the symptoms of the disease.
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 with autoimmune haemolytic anaemia were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for autoimmune haemolytic anaemia 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 21 January 2016 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
- sutimlimab
- Intended use
- Treatment of autoimmune haemolytic anaemia
- Orphan designation status
- Positive
- EU designation number
- EU/3/16/1609
- Date of designation
- Sponsor
Recordati Rare Diseases
Review of designation
The Committee for Orphan Medicinal Products reviewed the orphan designation of Enjaymo at the time of marketing authorisation, and confirmed that the orphan designation should be maintained. More information is available in the Enjaymo : Orphan maintenance assessment report (initial authorisation)
Update history
| Date | Update |
|---|---|
| February 2025 | The sponsorship was transferred to Recordati Rare Diseases. |
| July 2023 | The sponsor’s name was changed from Genzyme Europe B.V. to Sanofi B.V. in July 2023. |
| November 2019 | The sponsorship was transferred to Genzyme Europe B.V., Netherlands. |
| January 2019 | The sponsorship was transferred to Celerion Austria GmbH, Austria. |
| November 2018 | The sponsor changed name to Celerion United Kingdom Limited. |
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: