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Orphan designation

On 19 January 2009, orphan designation (EU/3/08/602) was granted by the European Commission to Immunomedics GmbH, Germany, for milatuzumab for the treatment of chronic lymphocytic leukaemia.

What is chronic lymphocytic leukaemia?

Chronic lymphocytic leukaemia (CLL) 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 is the estimated number of patients affected by the condition?

At the time of designation, CLL affected approximately 3.5 in 10,000 people in the European Union (EU)*. This is based on the information provided by the sponsor and knowledge of the Committee for Orphan Medicinal Products (COMP). This is below the threshold for orphan designation which is 5 in 10,000. This is equivalent to a total of around 175,800 people.

*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 27), Norway, Iceland and Liechtenstein. This represents a population of 502,282,000 (Eurostat 2008).

What treatments are 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). At the time of submission of the application for orphan drug designation, several products were authorised for the condition in the Community.

The sponsor has provided sufficient information to show that milatuzumab might be of significant benefit for the patients because it might act differently from other medicinal products, as it uses a different mechanism of action. 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?

Milatuzumab is a monoclonal antibody (a type of protein) that has been designed to recognise and bind to a specific structure (called an antigen) called CD74. This is a receptor protein that is often found on the surface of chronic lymphocytic leukaemia cells and is involved in the growth and survival of the cells. By attaching itself to the CD74 on cancer cells, milatuzumab is expected to stop their development and cause the cells to die.

What is the stage of development of this medicine?

The effects of milatuzumab have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials in patients with chronic lymphocytic leukaemia were ongoing.

At the time of submission, milatuzumab was not authorised anywhere in the world for chronic lymphocytic leukaemia. Orphan designation of milatuzumab had been granted in the United States for treatment of chronic lymphocytic leukaemia.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 5 November 2008 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;
  • and either the rarity of the condition (affecting not more than five in 10,000 people in the Community) or the 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 the quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.

Key facts

Product details for <p>Milatuzumab</p>
Active substanceMilatuzumab
Medicine Name
Disease/conditionTreatment of chronic lymphocytic leukaemia
Date of decision18/01/2009
Orphan decision numberEU/3/08/602

Review of designation

Sponsor’s contact details

Immunomedics GmbH
Paul-Ehrlich-Str. 22/D1
63322 Rödermark
Tel. +49 61 51 66 715 66
Fax + 49 6151 66 715 77

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.