EU/3/12/962

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Orphan designation

On 22 May 2012, orphan designation (EU/3/12/962) was granted by the European Commission to Gregory Fryer Associates Ltd, United Kingdom, for chimeric monoclonal antibody against kappa myeloma antigen 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 less than 2.9 in 10,000 people in the European Union (EU)*. This is equivalent to a total of fewer than 147,000 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 defenses. 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 chimeric monoclonal antibody against kappa myeloma antigen might be of significant benefit for patients with multiple myeloma because it works in a different way to existing treatments, and may be used in combination with other medicines to improve the outcome of patients with this condition. These assumptions 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?

Chimeric monoclonal antibody against kappa myeloma antigen is a monoclonal antibody (a type of protein) that has been designed to recognise and attach to a specific structure (an antigen) on the myeloma cell called ‘kappa myeloma antigen’. Once attached, it is expected to activate cells of the immune system which will kill the cancerous plasma cells.

What is the stage of development of this medicine?

The effects of chimeric monoclonal antibody against kappa myeloma antigen 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 chimeric monoclonal antibody against kappa myeloma antigen 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 January 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.

Key facts

Product details for <p>Chimeric monoclonal antibody against kappa myeloma antigen</p>
Active substanceChimeric monoclonal antibody against kappa myeloma antigen
Medicine Name
Disease/conditionTreatment of multiple myeloma
Date of decision22/05/2012
OutcomePositive
Orphan decision numberEU/3/12/962

Review of designation

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

Sponsor's contact details

Gregory Fryer Associates Ltd
30 St Thomas Place
Cambridgeshire Business Park
Ely
Cambridgeshire
CB7 4EX
United Kingdom
Telephone: +44 13 53 64 55 90
Telefax: Fax +44 13 53 64 55 99
E-mail: info@gf-associates.co.uk

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases see:

  • Orphanet, a database containing information on rare diseases which includes a directory of patients’ organisations registered in Europe.
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.