EU/3/12/1037 - orphan designation for treatment of multiple myeloma

Elotuzumab

Overview

Please note that this product (marketed as Empliciti) was withdrawn from the Community Register of designated Orphan Medicinal Products by the European Commission in April 2016, at the time of the granting of a marketing authorisation.

On 9 August 2012, orphan designation (EU/3/12/1037) was granted by the European Commission to Bristol-Myers Squibb Pharma EEIG, United Kingdom, for elotuzumab for the treatment of multiple myeloma.

What is multiple myeloma?

Multiple myeloma is a cancer of a type of white blood cells called plasma cells. Plasma cells are found 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 because it disrupts the normal functioning of the bone marrow, leads to bone destruction and causes kidney failure.

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

At the time of designation, multiple myeloma affected approximately 1.6 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 81,000 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 27), Norway, Iceland and Liechtenstein. This represents a population of 506,300,000 (Eurostat 2011).

What treatments are available?

At the time of submission of the application for orphan designation, several medicines were already authorised for multiple myeloma in the EU. The main treatment for multiple myeloma was chemotherapy (medicines to treat cancer) usually combined with steroids 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 and weakened bones. Interferon alfa, a protein normally produced by the body during viral infections, was sometimes used in combination with chemotherapy.

The sponsor has provided sufficient information to show that elotuzumab might be of significant benefit for patients with multiple myeloma because it targets a specific protein found in high levels on the surface of the cancer cells, and preliminary studies indicated that when used in combination with other medicines it may improve the outcome of patients with this condition. 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?

Elotuzumab is a monoclonal antibody, a type of protein that has been designed to recognise and attach to a specific structure in the body. Elotuzumab is expected to attach to a protein called human CD-2 subset 1 (CS1) that is found at high levels on the surface of myeloma cells.

When elotuzumab attaches to CS1 on the surface of myeloma cells, it is expected to activate certain cells of the immune system, called natural killer cells. These cells will then attach to the antibody and kill the cancer cells. This is expected to slow down the growth or cause the shrinkage of multiple myeloma tumours.

What is the stage of development of this medicine?

The effects of elotuzumab have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials in patients with multiple myeloma were ongoing.

At the time of submission, elotuzumab was not authorised anywhere in the world for multiple myeloma. Orphan designation of elotuzumab had been granted in the United States of America for multiple myeloma.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 11 July 2012 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.

EU/3/12/1037: Public summary of opinion on orphan designation: Elotuzumab for the treatment of multiple myeloma

Adopted Reference Number: EMA/COMP/528459/2012

Key facts

• Active substance:
  Elotuzumab
• Intended use:
  Treatment of multiple myeloma
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/12/1037
• Date of designation:
  09/08/2012
• Sponsor:

  Bristol-Myers Squibb Pharma EEIG;

Review of designation

On 8 April 2016, the Committee for Orphan Medicinal Products (COMP) completed its review of the designation EU/3/12/1037 of Empliciti (elotuzumab) as an orphan medicinal product for the treatment of multiple myeloma. The COMP assessed whether, at the time of marketing authorisation, the medicinal product still met the criteria for orphan designation. The Committee looked at the seriousness and prevalence of the condition, and the existence of other methods of treatment. As other methods of treatment are authorised in the European Union (EU), the COMP also considered whether the medicine is of significant benefit to patients with multiple myeloma. The COMP concluded that one of the criteria for orphan designation was no longer met and therefore recommended that the orphan designation of the product should not be maintained¹.

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¹The removal of the orphan designation at time of marketing authorisation means that the product cannot benefit from 10 years of market exclusivity in the EU. This means that in the 10 years after its authorisation similar products with the same therapeutic indication can be placed on the market.