EU/3/03/136

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Orphan designation

On 14 February 2003, orphan designation (EU/3/03/136) was granted by the European Commission to Amersham plc, U.K., for iodine (131I) tositumomab for the treatment of follicular lymphoma.

The sponsorship was transferred to GlaxoSmithKline Research & Development Limited, United Kingdom, in August 2005.

What is follicular lymphoma?

Lymphoma is a disease in which malignant (cancer) cells form in the lymphatic system. The lymphatic system consists of the tissues and organs that produce, store, and carry white blood cells. These cells fight infection and other diseases. There are several different types of lymphomas. In follicular lymphoma, the cancer cells are related to white blood cells called B-lymphocytes. Follicular lymphoma is a life-threatening disease.

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

At the time of designation, follicular lymphoma affected approximately 3.6 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 136,000 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: The number of patients affected by the condition is estimated and assessed for the purpose of the designation, for a European Community population of 377,000,000 (Eurostat 2001) and may differ from the true number of patients affected by the condition. This estimate is based on available information and calculations presented by the sponsor at the time of the application.

What treatments are available?

At the time of submission of the application for orphan drug designation, several medicinal products were authorised in the European Union for use in follicular lymphoma. The choice of treatment depends in particular on the extension of the disease as well as on the responses to therapies previously prescribed. Iodine (131I) tositumomab might be of potential significant benefit for the treatment of follicular lymphoma in association with tositumomab, particularly in terms of efficacy in advanced stages. The assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

How is this medicine expected to work?

Iodine (131I) tositumomab is an antibody which can carry a radioactive particle (iodine-131). Antibodies are proteins which specifically recognise and attach themselves to certain foreign substances, such as proteins found on the surface of cancer cells or bacteria. In the case of tositumomab, the antibody targets a specific structure on B-cell lymphocytes involved in follicular lymphoma called the CD20 receptor. The antibody is expected to bind to the cancer cells and to kill them with the radiation from the iodine.

What is the stage of development of this medicinal product?

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

Iodine (131I) tositumomab in association with tositumomab was granted orphan drug status in the United States of America on 16 May 1994 for the indication of treatment of B-cell non-Hodgkin’s lymphoma. 

Iodine (131I) tositumomab was not marketed anywhere worldwide for follicular lymphoma, at the time of submission.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 10 January 2003 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>Iodine (<sup>131</sup>I) tositumomab</p>
Active substanceIodine (131I) tositumomab
Medicine Name
Disease/conditionTreatment of follicular lymphoma
Date of decision13/02/2003
OutcomePositive
Orphan decision numberEU/3/03/136

Review of designation

Sponsor’s contact details:

GlaxoSmithKline Research & Development Ltd
1-3 Iron Bridge Road
Stockley Park West
Middlesex UB11 1BT
United Kingdom
Telephone: 44 208 990 38 79
Telefax: +44 208 990 35 11

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.