EU/3/14/1423: Orphan designation for the treatment of plasma cell myeloma

Plasma cell myeloma (also called multiple myeloma) is a cancer of a type of white blood cell called plasma cells. Plasma cells originate from the bone marrow, the spongy tissue inside the large bones in the body. In plasma cell 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 the 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.

Plasma cell 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, plasma cell myeloma affected approximately 3.6 in 10,000 people in the European Union (EU). This was equivalent to a total of around 184,000 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 511,100,000 (Eurostat 2014).

At the time of designation, several medicines were already authorised for plasma cell myeloma in the EU. The main treatment for plasma cell 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 an allogeneic stem-cell transplant (a complex procedure where the patient receives stem cells from a matched donor to help restore the bone marrow). Radiotherapy (using radiation to kill cancer cells) was used to treat pain due to bone damage and prevent further damage. Interferon alfa was sometimes used in combination with chemotherapy.

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with plasma cell myeloma because it works in a different way to existing treatments and early studies have shown that it might be beneficial for those patients who had cancer cells left in the body despite previous treatment. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

Human mucin-1 is a protein that is found on the surface of the cancerous cells in plasma cell myeloma but is rarely found on normal plasma cells. A large part of the mucin-1 protein is released into the blood. This medicine is made up of the short part of human mucin-1, which appears only on the cancer cells but not in the blood. When this medicine is given to patients, the immune system (the body's natural defences) is expected to recognise it as 'foreign'. This is expected to stimulate an immune response against human mucin-1 on the cancer cells, resulting in the immune system attacking and killing them.

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 plasma cell myeloma were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for plasma cell myeloma 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 11 December 2014 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.