EU/3/09/642

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Orphan designation

On 12 June 2009, orphan designation (EU/3/09/642) was granted by the European Commission to Centocor B.V., the Netherlands, for chimeric-anti-interleukin-6 monoclonal antibody for the treatment of multiple myeloma.

Centocor B.V. changed its name to Janssen Biologisc B.V. in July 2011.

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 life-threatening disease that leads to poor long-term survival.

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

At the time of designation, multiple myeloma affected approximately 2.2 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 11,000 people, and is below the threshold for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and 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 504,800,000 (Eurostat 2009).

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 is chemotherapy (medicines to treat cancer) usually combined with steroids (medicines used to reduce the activity of the immune system). Treatment with radiotherapy (using radiation to kill cancer cells) can be very useful in treating pain and weakened bones. Interferon alfa, a protein normally produced by the body during viral infections, can sometimes be used in combination with chemotherapy.
The sponsor has provided sufficient information to show that chimeric-anti-interleukin-6 monoclonal antibody 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 their effectiveness. 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-anti-interleukin-6 monoclonal antibody is a monoclonal antibody (a type of protein) that has been designed to recognise and attach to a specific structure (an antigen) called interleukin-6 (IL-6). IL-6 is a protein in the immune system (the body’s natural defences) that plays a role in the growth, survival and spread of cancerous plasma cells in multiple myeloma. By attaching to IL-6, the monoclonal antibody is expected to block the protein’s activity, slowing down the growth and spread of the cancer.

What is the stage of development of this medicine?

The effects of chimeric-anti-interleukin-6 monoclonal antibody 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, this medicine was not authorised anywhere in the EU for multiple myeloma.

Orphan designation of this medicine 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 5 May 2009 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 Community) 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 anti-interleukin-6 monoclonal antibody</p>
Active substanceChimeric anti-interleukin-6 monoclonal antibody
Medicine Name
Disease/conditionTreatment of multiple myeloma
Date of decision12/06/2009
OutcomePositive
Orphan decision numberEU/3/09/642

Review of designation

Sponsor’s contact details:

Janssen Biologisc B.V. 
Einsteinweg 101 
2333 CB Leiden 
The Netherlands 
Telephone: +31 71 524 22 27 
Telefax: +31 71 524 2493
E-mail: mbancsi@ITS.jnj.com

Patient associations’ contact points:

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.