EU/3/02/089 - orphan designation for treatment of juvenile myelomonocytic leukaemia

GM-CSF receptor antagonist

Overview

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in September 2002 on request from the sponsor.

On 18 March 2002, orphan designation (EU/3/02/089) was granted by the European Commission to British Biotech Pharmaceuticals Ltd, United Kingdom, for granulocyte macrophage colony stimulating factor (GM-CSF) receptor antagonist for the treatment of juvenile myelomonocytic leukaemia (JMML).

What is juvenile myelomonocytic leukaemia?

When leukaemia develops, the body produces large numbers of abnormal blood cells. There are several types of leukaemias. They are grouped by how quickly the disease develops and by the type of blood cell that is affected. JMML is a life-threatening disorder of early childhood.

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 patient's age, symptoms, and general health. Current treatments for JMML include bone marrow transplantation and chemotherapy. Some anticancer agents were authorised in the Community for the treatment of leukaemia at the time of submission of the application for orphan designation. Satisfactory argumentation has been submitted by the sponsor to justify the assumption that GM-CSF might be of potential significant benefit for the treatment of JMML, particularly in terms of its novel mechanism of action and the activity shown in pre-clinical studies.

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

According to the information provided by the sponsor, JMML was considered to affect about 400 persons in the European Union at the time of submission of the application for orphan medicinal product designation.

---

*Disclaimer: The number of patients affected by the condition is estimated and assessed for the purpose of the designation, for a European Community population of 377,000,000 (Eurostat 2001) and may differ from the true number of patients affected by the condition. 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? 

GM-CSF is a growth factor which regulates processes such as the production and activation of mature blood cells. JMML cells are particularly sensitive to this growth factor. The GM-CSF receptor antagonist is a modified form of growth factor that antagonises the action of the natural growth factor and this can result in the inhibition of the survival and proliferation of JMML cells.

What is the stage of development of this medicinal product?

At the time of submission of the application for orphan designation, clinical trials in patients with JMML had not been initiated.

GM-CSF had not been marketed anywhere worldwide for JMML or designated as an 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 23 January 2002 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 be affecting 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 have been 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.
GM-CSF receptor antagonist had not been marketed or designated as orphan medicinal product elsewhere, at the time of submission.

EU/3/02/089: Public summary of positive opinion for orphan designation of granulocyte macrophage colony stimulating factor receptor antagonist for the treatment of juvenile myelomonocytic leukaemia

Adopted Reference Number: EMEA/COMP/67/02 Rev. 2

Key facts

• Active substance:
  GM-CSF receptor antagonist
• Intended use:
  Treatment of juvenile myelomonocytic leukaemia
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/02/089
• Date of designation:
  18/03/2002
• Sponsor:

  British Biotech Pharmaceuticals Ltd; Watlington Road; Oxford OX4 6LY; United Kingdom; Tel. [phone]; Fax [phone]; E-mail: [e-mail]