EU/3/17/1900: Orphan designation for the treatment of myelodysplastic syndromes

Myelodysplastic syndromes are a group of disorders in which the red blood cells, white blood cells and platelets produced by the bone marrow (the spongy tissue inside large bones) do not mature normally. Patients with myelodysplastic syndromes can develop several symptoms including tiredness or weakness due to anaemia (low red blood cell counts), infections due to low white blood cell counts, and bruising or abnormal bleeding due to low platelet counts.

Myelodysplastic syndromes are long-term debilitating and life-threatening diseases because they can lead to severe anaemia, infections or bleeding, and can result in leukaemia (cancer of the white blood cells).

At the time of designation, myelodysplastic syndromes affected less than 2 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 103,000 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 515,700,000 (Eurostat 2017).

At the time of designation, some medicines were authorised in the EU for the treatment of myelodysplastic syndromes. The choice of treatment depended on a number of factors, including the type and the extent of the disease, whether it had been treated before, and the patient's age, symptoms and general state of health. The main treatments included medicines that stimulate production of blood cells, chemotherapy (medicines to treat cancer), blood transfusions and stem cell transplantation. Stem cell transplantation is a procedure where the patient's bone marrow is cleared of cells and replaced with stem cells from a donor to form new bone marrow that produces healthy blood cells.

The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with myelodysplastic syndromes. Observational data showed that the medicine led to improvements in patients with low or intermediate risk disease who are not specifically targeted by current treatments.

This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

Asunercept works by attaching to a protein known as the CD95 ligand, which triggers a natural process that leads to cell death (apoptosis). The CD95 ligand plays an important role in controlling the production of blood cells and is found at excess levels in the blood cells of patients with myelodysplastic syndromes. By attaching to the protein, the medicine is expected to block the triggering of apoptosis, allowing blood cells to grow and mature normally.

The effects of asunercept have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with asunercept in patients with myelodysplastic syndromes were ongoing.

At the time of submission, asunercept was not authorised anywhere in the EU for myelodysplastic syndromes. Orphan designation had been granted in the United States for the condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 13 July 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.