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Orphan designation

On 1 December 2003, orphan designation (EU/3/03/175) was granted by the European Commission to Micromet AG, Germany, for recombinant antibody derivative against human CD19 and CD3 for the treatment of mantle-cell lymphoma.

In January 2012, Micromet AG changed name to Micromet GmbH. In May 2012, Micromet GmbH changed name to Amgen Research (Munich) GmbH.

The sponsorship was transferred to Amgen Europe BV, The Netherlands, in February 2014.

For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.

What is mantle-cell lymphoma?

Mantle-cell lymphoma belongs to the group of non-Hodgkin's lymphoma. Lymphoma is a type of cancer that originates from the lymphatic system. The lymphatic system is part of the immune system, the body's natural defence against infection and disease. It is a complex system made up of organs such as bone marrow (the spongy tissue inside the large bones in the body), the thymus and the spleen, and a network of lymph nodes throughout the body that are connected by lymphatic vessels.

Normally, the lymphatic cells grow in a controlled manner. However, in lymphoma a lymphatic cell clone continues to grow and divides in an uncontrolled manner, developing into a tumour. Lymphoma cells generally grow in lymph nodes. There are several different types of lymphoma depending on the type of lymphatic cancer cells. In mantle-cell lymphoma, the cancer cells are related to the blood cells called B lymphocytes. This disease affects mainly adult males of over 50 years. Mantle-cell lymphoma is a serious and life-threatening condition.

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

At the time of designation, mantle-cell lymphoma affected approximately 0.4 in 10,000 people in the European Union (EU). This was equivalent to a total of 15,000 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. At the time of designation, this represented a population of 382,800,000 (Eurostat 2003).

What are the methods of treatment available?

Treatment of mantle-cell lymphoma includes chemotherapy (using drugs to kill cancer cells), radiotherapy (using high-dose X-rays or other high-energy rays to kill cancer cells) and immunotherapy (using drugs that stimulate the body’s own immune system to kill the cancer cells). 

There are currently several medicinal products authorised in the Community for treatment of non-Hodgkin lymphomas. The choice of treatment depends in particular on the extension of the disease as well as on the responses to therapies previously prescribed. Recombinant antibody derivative against human CD19 and CD3 could be of potential significant benefit for the treatment of mantle-cell lymphoma. The potential benefit is mainly because it may act in a different way to other drugs. This assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

How is this medicinal product expected to act?

Antibodies are proteins in the body that target and link specific shapes on the surface of foreign bodies, such as bacteria or cancer cells. Recombinant antibody derivative against human CD19 and CD3 is an antibody that is thought to bind to lymphoma cells (that display a protein called CD19 on the surface) as well as to cells of the immune system called T lymphocytes (that display CD3). By binding both these cells, it is thought that the antibody may help the body's immune system to recognise attack and kill the cancer cells.

What is the stage of development of this medicinal product?

The effects of recombinant antibody derivative against human CD19 and CD3 have been evaluated in experimental models. At the time of submission of the application for orphan designation, phase-I clinical trials including patients with mantle-cell lymphoma were ongoing. 

Recombinant antibody derivative against human CD19 and CD3 was not marketed anywhere worldwide for mantle-cell lymphoma or designated as orphan medicinal product elsewhere for this condition, 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 October 2003 recommending the granting of this designation.

Opinions on orphan-medicinal-product designations are based on the following cumulative 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>Recombinant antibody derivative against human CD19 and CD3</p>
Active substanceRecombinant antibody derivative against human CD19 and CD3
Medicine Name
Disease/conditionTreatment of mantle-cell lymphoma
Date of decision01/12/2003
Orphan decision numberEU/3/03/175

Review of designation

Sponsor’s contact details

Amgen Europe BV
Minervum 7061
4817 ZK Breda
The Netherlands
Tel. +31 765732000
Fax: +31 765732001

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.