EU/3/04/249

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Orphan designation

On 23 December 2004, orphan designation (EU/3/04/249) was granted by the European Commission to CellGenix Technologie Transfer GmbH, Germany, for recombinant histidine tagged idiotype immunoglobulin Fab fragment of clonal B-cell receptors for the treatment of follicular lymphoma.

CellGenix Technologie Transfer GmbH has changed its name to CellGenix GmbH in March 2011.

What is follicular lymphoma?

Follicular lymphoma is a type of cancer of the lymphatic system. The lymphatic system is part of the body’s immune system and helps fighting infections. It is a complex system made up of organs such as the bone marrow, the thymus (a gland behind the breast bone), the spleen (an organ in the abdomen, near the stomach), and the lymph nodes (or lymph glands, located throughout the body), which are connected by a network of tiny lymphatic vessels. There are two main types of cells, which make up the lymphatic tissue. These cells are called lymphocytes and belong to the group of white blood cells. The two types are called B lymphocytes (B cells) and T lymphocytes (T cells). There are several different types of lymphomas. In follicular lymphoma, the cancer cells are related to the Blymphocytes. 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 3.5 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 161,000 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 based on data from the European Union (EU 25), Norway, Iceland and Lichtenstein. This represents a population of 459,700,000 (Eurostat 2004). 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.

Recombinant histidine tagged idiotype immunoglobulin Fab fragment of clonal B-cell receptors might be of potential significant benefit for the treatment of follicular lymphoma because it may act in a different way than other available medicines and it might improve the long-term outcome of the patients. 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?

Recombinant histidine tagged idiotype immunoglobulin Fab fragment of clonal B-cell receptors is a vaccine that contains proteins and originate from the cancer cells from the patient him/herself. According to the sponsor, a specific protein from the surface of the cancer cells is isolated and manipulated in the laboratory to be further injected back into the patient. It is expected that following the injection of this “patient-specific vaccine”, the body’s defence system (immune system) will recognize this protein as a foreign body and will react against it by producing specific antibodies. It is expected that the immune system will not only recognize the vaccine as foreign, but also the cancer cells which carry on their surface the same protein as the one used to prepare the patient-specific vaccine. The patient body’s immune system could then kill these cancer cells. 

What is the stage of development of this medicine?

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

Recombinant histidine tagged idiotype immunoglobulin Fab fragment of clonal B-cell receptors was not marketed anywhere worldwide for follicular 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 11 November 2004 recommending the granting of this designation.

Opinions on orphan medicinal products 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 hisitidine-tagged idiotype immunoglobulin Fab fragment of clonal B-cell receptors</p>
Active substanceRecombinant hisitidine-tagged idiotype immunoglobulin Fab fragment of clonal B-cell receptors
Medicine Name
Disease/conditionTreatment of follicular lymphoma
Date of decision23/12/2004
OutcomePositive
Orphan decision numberEU/3/04/249

Review of designation

Sponsor’s contact details:

CellGenix GmbH
Am Flughafen 16
D-79108 Freiburg
Germany
Telephone: +49 761 88 88 90
Telefax: +49 761 88 88 98 00
E-mail: rosenthal@cellgenix.com

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.