EU/3/04/248

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Orphan designation

On 21 December 2004, orphan designation (EU/3/04/248) 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 multiple myeloma.

CellGenix Technologie Transfer GmbH has changed its name to CellGenix GmbH in March 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 bone marrow is the spongy tissue inside the large bones in the body. Normally, the bone marrow makes cells called “blasts” that mature into several different types of blood cells that have specific functions in the body. These include red cells, white cells and platelets. Red blood cells carry oxygen and other materials to all tissues of the body. Platelets make the blood clot, and white blood cells fight infection. In multiple myeloma an excessive number of plasma cells are produced. Normally, the division of cells takes place in a controlled manner but with multiple myeloma, the process gets out of control and abnormal plasma cells multiply, producing many myeloma cells. These fill up the bone marrow and interfere with production of the normal white cells, red cells and platelets. This leads to a number of possible complications, which include anaemia, bone pain and fractures, raised levels of calcium in the blood and kidney disease. Multiple myeloma is lifethreatening. 

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

At the time of designation, multiple myeloma affected approximately 1.2 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 55,000 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?

The main treatment of multiple myeloma is chemotherapy (using drugs to kill cancer cells) usually combined with steroids (a group of chemical substances, the so called hormones, which have an effect on the activity of certain organs). Other types of treatment for multiple myeloma are 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 cancer cells). Radiotherapy can be useful to treat painful areas and weakened bones. Several products were authorised for the condition in the Community at the time of submission of the application for orphan drug designation. 

Recombinant histidine tagged idiotype immunoglobulin Fab fragment of clonal B-cell receptors could be of potential significant benefit for the treatment of multiple myeloma because it may act in a different way than other available medicines. 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 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 multiple myeloma were ongoing.

Recombinant histidine tagged idiotype immunoglobulin Fab fragment of clonal B-cell receptors was not marketed anywhere worldwide for multiple myeloma 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 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 multiple myeloma
Date of decision20/12/2004
OutcomePositive
Orphan decision numberEU/3/04/248

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.