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Orphan designation

On 11 April 2005, orphan designation (EU/3/05/276) was granted by the European Commission to TeGenero AG, Germany, for humanised agonistic anti-CD28 monoclonal antibody for the treatment of B-cell chronic lymphocytic leukemia.

What is B-cell chronic lymphocytic leukemia?

B-cell chronic lymphocytic leukemia is a disease in which cancer cells are found in the blood and 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. White blood cells fight infection. Platelets support blood clotting. When leukemia develops, the bone marrow produces large numbers of abnormal blood cells. There are several types of leukemias. B-cell chronic lymphocytic leukemia is a cancer of the white blood cells called lymphocytes. The lymphocytes multiply too quickly and live too long, so there are too many of them circulating in the blood. These leukemic lymphocytes look normal, but they are not fully developed and do not work properly. Over a period of time these abnormal cells replace the normal white cells, red cells and platelets in the bone marrow. It is the most common type of leukemia. B-cell chronic lymphocytic leukemia mainly affects older people and is rare in people under the age of 40. Bcell chronic lymphocytic leukemia is  chronically debilitating and life-threatening due to the severe prognosis and the poor long-term survival for high-risk patients.

What are the methods of treatment available?

Treatment for leukemia is complex and depends on a number of factors including the type of leukemia, the extent of the disease and whether the leukemia has been treated before. It also depends on the age, the symptoms, and the general health of the patient. Some people with B-cell chronic lymphocytic leukemia never have treatment if their illness is not causing any symptoms and is progressing only very slowly. Treatment is only started if and when the symptoms become troublesome. Current main treatment of B-cell chronic lymphocytic leukemia is chemotherapy (using drugs to kill cancer cells). Several products were authorised for the condition in the Community at the time of submission of the application for orphan drug designation. Humanised agonistic anti-CD28 monoclonal antibody could be of potential significant benefit for the treatment of chronic lymphocytic leukemia because it may offer a new way of killing cancer cells. This assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status. 

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

According to the information provided by the sponsor, B-cell chronic lymphocytic leukemia was considered to affect about 124,000 persons in the European Union.

*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.

How is this medicinal product expected to act?

Antibodies are proteins in the body that target and link specific shapes (the so-called antigens) on the surface of, various cells. Humanised agonistic anti-CD28 monoclonal antibody is an antibody that is thought to bind a specific type of cells of the immune system called T lymphocytes. By binding these cells, it is thought that the antibody can stimulate them to attack and kill the cancer cells.

What is the stage of development of this medicinal product?

The evaluation of the effects of humanised agonistic anti-CD28 monoclonal antibody in experimental models is ongoing. At the time of submission of the application for orphan designation, no clinical trials in patients with Bcell chronic lymphocytic leukemia were initiated. Humanised agonistic anti-CD28 monoclonal antibody was not marketed anywhere worldwide for B-cell chronic lymphocytic leukemia or designated as orphan medicinal product elsewhere for this condition, a  the time of submission.
According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 3 March 2005 a positive opinion recommending the grant of the above-mentioned designation.

Opinions on orphan medicinal products designations are based on the following cumulative criteria:
  • the seriousness of the condition, 
  • the existence or not of alternative methods of diagnosis, prevention or treatment and 
  • either the rarity of the condition (considered to affect not more than five in ten thousand persons in the Community) or the insufficient return of development investments. 

Designated orphan medicinal products are still investigational products which were considered for designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of the quality, safety and efficacy will be necessary before this product can be granted a marketing authorisation. 

Key facts

Product details for <p>Humanised agonistic anti-CD28 monoclonal antibody</p>
Active substanceHumanised agonistic anti-CD28 monoclonal antibody
Medicine Name
Disease/conditionTreatment of B-cell chronic lymphocytic leukaemia
Date of decision11/04/2005
Orphan decision numberEU/3/05/276

Review of designation

Sponsor’s contact details:

TeGenero AG
Science Park Wuerzburg
Friedrich-Bergius-Ring 15
97076 Wuerzburg
Telephone: +49 93 13 59 62 0
Telefax: +49 93 13 59 62 11

Patients’ associations contact points:

Leukaemia Care
2 Shrubbery Avenue
Telephone: +44 19 05 33 00 03
Telefax: +44 19 05 33 00 90

Ligue Nationale Contre le Cancer
13 Av. de la Grande Armee
75116 Paris
Telephone: +33 1 45 00 00 17
Tefefax: +33 1 45 00 63 06

Associazione Italiana contro le Leucemie-linfomi e mieloma ONLUS
Via Ravenna, 34
00161 Roma
Telephone: +39 06 44 03 763
Telefax: +39 06 44 04 226