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Orphan designation

On 29 July 2004, orphan designation (EU/3/04/214) was granted by the European Commission to Novartis Europharm Limited, United Kingdom, for midostaurin for the treatment of acute myeloid leukaemia.

What is acute myeloid leukaemia?

Acute myeloid leukaemia 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 leukaemia develops, the bone marrow produces large numbers of abnormal blood cells. There are several types of leukaemias. In myeloid leukaemia blasts that are developing into 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. Leukaemia can be acute (when it develops quickly with many blasts). Acute myeloid leukaemia is life-threatening.

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 age, the symptoms, and the general health of the patient. The primary treatment of acute myeloid leukaemia 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. Midostaurin might be of potential significant benefit for the treatment of acute myeloid leukaemia, because it may act in a different way than other medicines and it might add to the effect of the current standard treatment. The 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 leukaemia was considered to affect about 29,000 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?

Enzymes are proteins produced by the human body that speed up the conversion of certain substances into other substances. Midostaurin inhibits an enzyme, the so-called fms-like tyrosine kinase 3 (FLT3). It has been found that this enzyme plays an important role in the survival and self-renewal of the blast cells, but not  anymore after the maturation of these cells. FLT3 has been identified in >80% of the myeloid leukaemia blast cells. Midostaurin might, by inhibition of this FLT3 enzyme activity, help in slowing down or stopping the further growth of the leukaemia cells and might help in the destruction of these cells carrying FLT3.

What is the stage of development of this medicinal product?

The effects of midostaurin were evaluated in experimental models. At the time of submission of the application for orphan designation, clinical trials in patients with acute myeloid leukaemia were ongoing. Midostaurin was not marketed anywhere worldwide for acute myeloid leukaemia or designated as 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 16 June 2004 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>Midostaurin</p>
Active substanceMidostaurin
Medicine Name
Disease/conditionTreatment of acute myeloid leukaemia
Date of decision29/07/2004
Orphan decision numberEU/3/04/214

Review of designation

Sponsor’s contact details:

Novartis Europharm Limited
Wimblehurst Road
West Sussex RH12 5AB
United Kingdom
Telephone: +44 14 03 27 28 27
Telefax: +44 14 03 32 30 60

Patients’ associations contact points:

Leukaemia Research Foundation
43 Great Ormond Street
United Kingdom
Telephone: +44 20 74 05 01 01

Leukaemia Care
2 Shrubbery Avenue
United Kingdom
0800 169 6680 (freephone for UK residents)
Telephone: +44 19 05 33 00 03 / + 44 84 57 67 32 03

Ligue Nationale Contre le Cancer
13 Av. de la Grande Armee
75116 Paris
Telephone: +33 1 45 00 00 17
Tefefax: +33 1 45 00 63 06