EU/3/16/1609: Orphan designation for the treatment of autoimmune haemolytic anaemia

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 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 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.