EU/3/17/1863: Orphan designation for the treatment of multiple myeloma

Multiple myeloma (also called plasma cell myeloma) is a cancer of a type of white blood cell called plasma cells. Plasma cells originate in the bone marrow, the spongy tissue inside the large bones in the body. In multiple myeloma, the division of plasma cells becomes out of control, resulting in abnormal, immature plasma cells multiplying and filling up the bone marrow. This interferes with production of normal white blood cells, red blood cells and platelets (components that help the blood to clot), leading to complications such as anaemia (low red blood cell counts), bone pain and fractures, raised blood calcium levels and kidney disease.

Multiple myeloma is a debilitating and life-threatening disease particularly because it disrupts the normal functioning of the bone marrow, damages the bones and causes kidney failure.

At the time of designation, multiple myeloma affected approximately 3.6 in 10,000 people in the European Union (EU). This was equivalent to a total of around 186,000 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, several medicines were authorised for multiple myeloma in the EU. The main treatment for multiple myeloma was chemotherapy (medicines to treat cancer) usually combined with corticosteroids to reduce the activity of the immune system, the body's natural defences. Where chemotherapy did not work, some patients received a stem-cell transplant (a procedure where the patient's bone marrow is replaced with stem cells to form new bone marrow that produces healthy blood cells). Radiotherapy (using radiation to kill cancer cells) was used to treat pain due to bone damage and prevent further damage.

The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with multiple myeloma because early studies indicated that patients whose disease came back after previous treatment responded to treatment with this medicine. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

The abnormal immature plasma cells in patients with multiple myeloma produce a protein on their surface called B-cell maturation antigen (BCMA).

This medicine is made up of T cells (a type of white blood cell) which are taken from the patient. The T cells are modified in the laboratory with a virus that carries a gene into the T cells so that they can recognise and attach to BCMA. The T cells are then given back to the patient, where they are expected to attach to BCMA on the cancer cells and kill them, and to activate other T cells.

The type of virus used in this medicine ('lentivirus') is modified in order not to cause disease in humans.

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 multiple myeloma were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for multiple myeloma. Orphan designation of the medicine had been granted in the United States for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 15 March 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.