EU/3/20/2292: Orphan designation for the treatment of blastic plasmacytoid dendritic cell neoplasm

Blastic plasmacytoid dendritic cell neoplasm is an aggressive type of blood cancer in which the bone marrow (the spongy tissue inside the large bones, where blood cells are produced) produces large numbers of immature white blood cells called 'plasmacytoid dendritic cells'. These may build up in the bone marrow and take the place of normal blood cells. Most patients develop non-itchy damaged areas in the skin, which often look like bruises or nodules. The disease may also cause enlargement of the spleen or liver and a reduction of the number of circulating blood cells.

Blastic plasmacytoid dendritic cell neoplasm is a life-threatening disease that is associated with poor overall survival.

At the time of designation, blastic plasmacytoid dendritic cell neoplasm affected approximately 0.01 in 10,000 people in the European Union (EU). This was equivalent to a total of around 500 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).

*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, Iceland, Liechtenstein, Norway and the United Kingdom. This represents a population of 519,200,000 (Eurostat 2020).

At the time of designation, no satisfactory methods were authorised in the EU for the treatment of blastic plasmacytoid dendritic cell neoplasm. Patients often received radiotherapy (treatment with radiation) and medicines authorised for other types of blood cancers. Some patients were also given stem cell transplantation, a complex procedure where the patient receives stem cells from a matched donor to help restore the bone marrow.

This medicine is made of an antibody (a type of protein) linked to a substance that kills cancer cells. The antibody portion of the medicine attaches to a receptor (target) called CD123, which is found at high levels on the surface of cancerous plasmacytoid dendritic cells. Once attached to the receptor, the medicine is taken up inside the cell where the toxic portion interferes with the production of the cell’s DNA (genetic material), causing the death of the cell. This is expected to slow down the progression 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 blastic plasmacytoid dendritic cell neoplasm were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for the treatment of blastic plasmacytoid dendritic cell neoplasm or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 20 May 2020, 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.