EU/3/09/656

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Orphan designation

On 24 July 2009, orphan designation (EU/3/09/656) 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 diffuse large B-cell lymphoma.

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

What is diffuse large B-cell lymphoma?

Diffuse large B-cell lymphoma is the most common cancer of the lymphatic system, a network of vessels that transport lymph from tissues through the lymph nodes and into the bloodstream. Diffuse large B-cell lymphoma affects a type of white blood cell called B-lymphocytes, or B cells. In diffuse large B-cell lymphoma, the B cells multiply too quickly and live for too long, so there are too many of them in the lymph nodes. The disease first shows as a lump in the neck, under the arm or in the groin area, caused by an enlarged lymph node. Patients with diffuse large B-cell lymphoma may have unexplained fever, tiredness, night sweats and weight loss.
Although some people with diffuse large B-cell lymphoma can be cured, it remains a serious and life-threatening disease, particularly when the disease is found late or has come back after initial treatment.

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

At the time of designation, diffuse large B-cell lymphoma affected approximately 2 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 101,000 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 designation, several medicines were authorised for the treatment of diffuse large B-cell lymphoma in the EU. The main treatment was chemotherapy (medicines to treat cancer), sometimes in combination with radiotherapy (treatment with radiation). Bone marrow transplantation was also used in patients at risk of the disease coming back after treatment. This is a complex procedure where the bone marrow of the patient is destroyed and replaced with healthy bone marrow from the same patient or a matched donor.

The sponsor has provided sufficient information to show that recombinant histidine-tagged idiotype immunoglobulin Fab fragment of clonal B-cell receptors might be of significant benefit for patients with diffuse large B-cell lymphoma because it works in a different way to existing treatments and could be an alternative treatment for this disease in patients with limited or no other treatment options.
This assumption 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?

Recombinant histidine-tagged idiotype immunoglobulin Fab fragment of clonal B-cell receptors is a cancer vaccine. It is expected to work by activating the patient’s immune system (the body’s natural defences) so that it attacks and kills the cancer cells. To prepare this medicine, tumour cells are extracted from the patient. The cells are analysed in the laboratory to identify ‘idiotypes’, proteins that are found on the surface of the cancer cells and that are specific to the patient’s cancer cells. These proteins are then grown in the laboratory before being put into a vaccine together with other components that will help the immune system to recognise the proteins from the cancer cells as ‘foreign’ when the vaccine is injected back into the patient. This is expected to stimulate an immune response against the cancer cells.

What is the stage of development of this medicine?

The effects of this medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials in patients with diffuse large B-cell lymphoma had been started.

At the time of submission, this medicine was not authorised anywhere in the EU for diffuse large B-cell lymphoma or designated as orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 4 June 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>Recombinant histidine-tagged idiotype immunoglobulin Fab fragment of clonal B-cell receptors</p>
Active substanceRecombinant histidine-tagged idiotype immunoglobulin Fab fragment of clonal B-cell receptors
Medicine Name
Disease/conditionTreatment of diffuse large B-cell lymphoma
Date of decision24/07/2009
OutcomePositive
Orphan decision numberEU/3/09/656

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.