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Orphan designation

On 10 March 2005, orphan designation (EU/3/05/269) was granted by the European Commission to Janssen-Cilag International N.V., Belgium, for tipifarnib for the treatment of acute myeloid leukaemia.

The sponsorship was transferred to TMC Pharma Services Ltd, United Kingdom, in June 2015.

What is acute myeloid leukemia?

Acute myeloid leukemia 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 make the blood clot. When leukemia develops, the bone marrow produces large numbers of abnormal blood cells. There are several types of leukemias. In myeloid leukemia blasts that are developing into a type of white blood cells called granulocytes are affected. The blasts do not mature and become too many. These blast cells are then found in the blood and also accumulate in the bone marrow where they take the place of the other types of normal blood cells. Leukemia can be acute (when it develops quickly with many blasts). Acute myeloid leukemia is life-threatening.

What are the methods of treatment available?

Treatment for leukemia 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. The primary treatment of acute myeloid leukemia 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. Tipifarnib could be of potential significant benefit for the treatment of acute myeloid leukemia because it might improve the long-term outcome of the patients. 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*?

According to the information provided by the sponsor, acute myeloid leukemia was considered to affect about 32,200 persons in the European Union.

*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?

Tipifarnib is a molecule that represses or prevents the functioning of another molecule (the so-called farnesyl transferase). Farnesyl transferase is an enzyme (a protein that speed up the conversion of certain substances into other substances) indirectly involved in the reproduction and maturation of the 
leukemic cells. It is assumed that by blocking farnesyl transferase, tipifarnib might block the cancer cell division in acute myeloid leukemia.

What is the stage of development of this medicinal product?

The effects of tipifarnib were evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials in patients with acute myeloid leukemia were ongoing. Tipifarnib was not marketed anywhere worldwide for acute myeloid leukemia at the time of submission. Orphan designation of tipifarnib was granted in United States for treatment of acute myeloid leukemia. According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 3 February 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.

Key facts

Product details for <p>Tipifarnib</p>
Active substanceTipifarnib
Medicine Name
Disease/conditionTreatment of acute myeloid leukaemia
Date of decision09/03/2005
Orphan decision numberEU/3/05/269

Review of designation

Sponsor’s contact details

TMC Pharma Services Ltd
Lodge Farm Barn
Elvetham Park Estate
Fleet Road
Hartley Wintney
Hampshire RG27 8AS
United Kingdom
Tel. +44 (0)1252 842255
Fax +44 (0)1252 842277

Patients’ associations contact points

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.