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Orphan designation

On 29 January 2010, orphan designation (EU/3/09/713) was granted by the European Commission to Immunomedics GmbH, Germany, for veltuzumab for the treatment of chronic lymphocytic leukaemia.

What is chronic lymphocytic leukaemia?

Chronic lymphocytic leukaemia (CLL) is cancer of a type of white blood cell called B-lymphocytes. In this disease, the lymphocytes multiply too quickly and live for too long, so that there are too many of them circulating in the blood. The cancerous lymphocytes look normal, but they are not fully developed and do not work properly. Over a period of time, the abnormal cells replace the normal white cells, red cells and platelets (components that help the blood to clot) in the bone marrow (the spongy tissue inside the large bones in the body).

CLL is the most common type of leukaemia and mainly affects older people. It is rare in people under the age of 40 years. CLL is a long-term debilitating and life-threatening disease because some patients develop severe infections.

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

At the time of designation, chronic lymphocytic leukaemia affected approximately 3.5 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 177,000 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?

Treatment for CLL is complex and depends on a number of factors, including the extent of the disease, whether it has been treated before, and the patient’s age, symptoms and general state of health. Patients whose CLL is not causing any symptoms or is only getting worse very slowly may not need treatment. Treatment for CLL is only started if symptoms become troublesome. At the time of designation, the main treatment for CLL was chemotherapy (medicines to treat cancer).

The sponsor has provided sufficient information to show that veltuzumab might be of significant benefit for patients with CLL because results from studies in experimental models and early results from clinical studies indicate that it may improve the treatment of patients with this condition. 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?

Veltuzumab is a monoclonal antibody (a type of protein) that has been designed to recognise and attach to a specific structure (an antigen) called CD20. This is a receptor that is found on the surface of all B-lymphocytes. When this medicine binds to CD20, it is expected to cause the death of the cancerous B-lymphocytes.

What is the stage of development of this medicine?

The effects of veltuzumab have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with the designated product in patients with CLL were ongoing.

At the time of submission, veltuzumab was not authorised anywhere in the EU for CLL. Orphan designation of veltuzumab had been granted in the United States of America for CLL.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 5 November 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>Veltuzumab</p>
Active substanceVeltuzumab
Medicine Name
Disease/conditionTreatment of chronic lymphocytic leukaemia
Date of decision29/01/2010
Orphan decision numberEU/3/09/713

Review of designation

Contact point:

Sponsor’s contact details:

Immunomedics GmbH
Otto-Röhm-Straße 69
64293 Darmstadt
Telephone: + 49 6151 66 715 66
Telefax: + 49 6151 66 715 77

Patient associations’ contact points:

Leukaemia CARE
One Birch Court
Blackpole East
Worcestershire WR3 8SG
United Kingdom
Telephone: +44 1905 755977 / +44 845 521 3456
Telefax: +44 1905 755166

Ligue Nationale Contre le Cancer
14 Rue Corvisart
75013 Paris
Telephone: +33 1 53 55 24 00
Telefax: +33 1 43 36 91 10

European Cancer Patient Coalition
ECPC Office
Am Rothenanger 1b
85521 Riemerling
Telephone: +49 89 628 36 807
Telefax: +49 89 628 36 808