EU/3/17/1837

On 27 February 2017, orphan designation (EU/3/17/1837) was granted by the European Commission to Opsona Therapeutics Ltd, Ireland, for humanised IgG4 monoclonal antibody to the human toll-like receptor type 2 (also known as OPN-305) for the treatment of myelodysplastic syndromes.

What are 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 grow and 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).

What is the estimated number of patients affected by the condition?

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 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 515,700,000 (Eurostat 2017).

What treatments are available?

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 because early studies show that it may increase blood cell counts in patients with less aggressive forms of myelodysplastic syndromes and who cannot take other medicines for the condition or where other medicines have not worked. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

The medicine is a monoclonal antibody (a type of protein) that has been designed to attach to another protein called toll-like receptor type 2 (TLR2). TLR2 is part of the immune system (the body’s natural defences) and may play a role in abnormal growth of blood cells. By attaching to TLR2, this medicine is expected to block its activity, thereby leading to normal blood cell growth.

What is the stage of development of this medicine?

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 myelodysplastic syndromes were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for myelodysplastic syndromes. Orphan designation of the medicine had been granted in the US for myelodysplastic syndromes.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 19 January 2017 recommending the granting of this designation.

Opinions on orphan medicinal product designations are based on the following three criteria:

• 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.

The Committee for Orphan Medicinal Products reviews the orphan designation of a product it it is approved for marketing authorisation.