EU/3/12/1083 - orphan designation for treatment chronic lymphocytic leukaemia

Humanised single-chain monoclonal antibody against CD37 (otlertuzumab)

Overview

This medicine is now known as otlertuzumab.

On 6 December 2012, orphan designation (EU/3/12/1083) was granted by the European Commission to Emergent Product Development UK Limited, United Kingdom, for humanised single-chain monoclonal antibody against CD37 for the treatment chronic lymphocytic leukaemia.

The sponsorship was transferred to Aptevo Europe Limited, United Kingdom, in March 2017, to Global Regulatory Ltd, United Kingdom, in July 2018 and to Voisin Consulting S.A.R.L., France, in January 2019.

Please note that this product was withdrawn from the Union Register of orphan medicinal products in March 2021 on request of the Sponsor.

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, CLL affected approximately 3 in 10,000 people in the European Union (EU). This is equivalent to a total of around 153,000 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).

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*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. At the time of designation, this represented a population of 509,000,000 (Eurostat 2012).

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 humanised single-chain monoclonal antibody against CD37 might be of significant benefit for patients with CLL because it works in a different way to existing treatments and early studies show that it might improve the outcome 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?

This medicine is a monoclonal antibody (a type of protein) that has been designed to recognise and attach to a specific structure (called an antigen) that is found on certain cells in the body. It has been designed to attach to CD37, which is present on the surface of B-lymphocytes. By attaching to CD37, the medicine is expected to cause the death of the cell. It is also expected to activate certain cells in the immune system (the body's natural defences), so that they kill the cancerous B-lymphocytes. This is expected to slow down the development of CLL.

What is the stage of development of this medicine?

The effects of humanised single-chain monoclonal antibody against CD37 have been evaluated in experimental models.

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

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

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 7 November 2012 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 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.

EU/3/12/1083 : Public summary of opinion on orphan designation: Humanised single-chain monoclonal antibody against CD37 for the treatment chronic lymphocytic leukaemia

Adopted Reference Number: EMA/COMP/741023/2012 Rev. 1

Key facts

• Active substance:
  Humanised single-chain monoclonal antibody against CD37 (otlertuzumab)
• Intended use:
  Treatment chronic lymphocytic leukaemia
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/12/1083
• Date of designation:
  06/12/2012
• Sponsor:

  Voisin Consulting S.A.R.L.; 64 Avenue Pierre Grenier; 92100 Boulogne-Billancourt; France; Tel. [phone]; E-mail: [e-mail]

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.