EU/3/05/306 - orphan designation for treatment of mastocytosis
imatinib mesilate
OrphanHuman
Overview
Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in November 2007 on request of the sponsor.
On 26 August 2005, orphan designation (EU/3/05/306) was granted by the European Commission to Novartis Europharm Limited, United Kingdom, for imatinib mesilate for the treatment of mastocytosis.
For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.
Mastocytosis is a disease in which a certain type of cells (so-called mast cells) accumulate excessively in the bone marrow and in other organs. 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 mastocytosis develops, large numbers of a certain white blood cell called mast cells are produced. In normal circumstances these cells are mainly located in the skin and in the linings of the intestine. Their role is two-fold: they take part in the defence of these tissues against diseases and they contribute in the development of allergic reactions.
The manifestation of mastocytosis is variable. In most of the patients, mainly in children, only the skin is involved and these lesions may spontaneously disappear (cutaneous mastocytosis). In some patients, more often occurring in adults, the cancer cells become aggressive tumours infiltrating organs (systemic mastocytosis) leading to organ failure and poor long-term outcome.
At the time of designation, mastocytosis affected less than 1 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 47,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).
*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 25), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 466,600,000 (Eurostat 2005).
No satisfactory methods exist that were authorised at the time of application.
Enzymes are proteins produced by the human body that speed up the conversion of certain substances into other substances. Imatinib mesilate blocks (inhibits) the enzyme tyrosine kinase. This enzyme plays a role in a cascade of molecular reactions to bring a certain signal from outside the cell into the cell thereby controlling the growth of the cells. In cancer cells, the function of this enzyme is disturbed causing uncontrolled growth and multiplication of the cancer cells. Imatinib mesilate might, by inhibition of this enzyme activity, help in slowing down or stopping the further growth of the cancer cells.
At the time of submission of the application for orphan designation, clinical trials in patients with mastocytosis were ongoing.
Imatinib mesilate was not marketed anywhere worldwide for mastocytosis, at the time of submission.
Orphan designation of imatinib mesilate was granted in Europe and in the United States for chronic myeloproliferative leukaemia and for gastrointestinal stromal tumours.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 13 July 2005 recommending the granting of this designation.
- 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
- Active substance
- imatinib mesilate
- Intended use
- Treatment of mastocytosis
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/05/306
- Date of designation
- Sponsor
Novartis Europharm Limited
EMA list of opinions on orphan medicinal product designation
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
Patients' organisations
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: