EU/3/05/347 - orphan designation for treatment of chronic lymphocytic leukaemia

human monoclonal antibody against HLA-DR

Overview

Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in July 2008 on request of the sponsor.

On 24 January 2006, orphan designation (EU/3/05/347) was granted by the European Commission to GPC Biotech AG, Germany, for human monoclonal antibody against HLA-DR for the treatment of chronic lymphocytic leukaemia.

What is chronic lymphocytic leukaemia?

Chronic lymphocytic leukaemia 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 leukaemia develops, the bone marrow produces large numbers of abnormal blood cells. There are several types of leukaemias. Chronic lymphocytic leukaemia is a cancer of a type of white blood cells called B-lymphocytes. The lymphocytes multiply too quickly and live too long, so there are too many of them circulating in the blood. These leukaemic 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. Chronic lymphocytic leukaemia is the most common type of leukaemia and mainly affects older people and is rare in people under the age of 40. Chronic lymphocytic leukaemia 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 leukaemia is complex and depends on a number of factors including the type of leukaemia, the extent of the disease and whether the leukaemia has been treated before. It also depends on the age, the symptoms, and the general health of the patient. Some people with chronic lymphocytic leukaemia do not 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 chronic lymphocytic leukaemia 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.
Human monoclonal antibody against HLA-DR might be of potential significant benefit for the treatment of chronic lymphocytic leukaemia because it might act differently from other medicinal products. 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*?

Based on the information provided by the sponsor and previous knowledge of the Committee, chronic lymphocytic leukaemia was considered to affect approximately 2.7 in 10,000 persons in the European Union, which, at the time of designation, corresponded to about 124,000 persons.

* 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 bind specific structures on the surface of foreign bodies, such as bacteria or cancer cells. Human monoclonal antibody against HLA-DR is an antibody that recognises a specific area of human leucocyte antigen-DR (HLA-DR), which is a protein present in the surface of some cells in the body, belonging to the immune system. In experimental conditions it has been shown that when the antibody binds the HLA-Dron malignant cells, it triggers their death Through this mechanism human monoclonal antibody against HLA-DR might induce the death of multiple myeloma cells.

What is the stage of development of this medicinal product?

The effects of human monoclonal antibody against HLA-DR were evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials including patients with chronic lymphocytic leukaemia were ongoing.
The medicinal product was not authorised anywhere worldwide for chronic lymphocytic leukaemia or designated as orphan medicinal product elsewhere for this condition, at the time of submission.
According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 7 December 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.

EU/3/05/347: Public summary of positive opinion for orphan designation of human monoclonal antibody against HLA-DR for the treatment of chronic lymphocytic leukaemia

Adopted Reference Number: EMEA/COMP/414041/2005 Rev.1

Key facts

• Active substance:
  human monoclonal antibody against HLA-DR
• Intended use:
  Treatment of chronic lymphocytic leukaemia
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/05/347
• Date of designation:
  24/01/2006
• Sponsor:

  GPC Biotech AG; Fraunhoferstrasse 20; D-82152 Martinsried; Munich; Germany; Telephone: [phone]; Telefax: [phone]; E-mail: [e-mail]