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Orphan designation

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in October 2011 on request of the sponsor.

On 23 March 2010, orphan designation (EU/3/10/729) was granted by the European Commission to Merck KGaA, Germany, for (1S, 2S, 3R, 4R)-3-(5-fluoro-2-(3-methyl-4-(4-methylpiperazin-1-yl)-phenylamino)-pyrimidin-4-ylamino)-bicyclo[2.2.1]hept-5-ene-2-carboxamide benzoate for the treatment of acute myeloid leukaemia.

What is acute myeloid leukaemia?

Acute myeloid leukaemia (AML) is a cancer of the white blood cells (cells that fight against infections). In patients with AML, the bone marrow (the spongy tissue inside the large bones) produces large numbers of abnormal, immature white blood cells called ‘blasts’. These abnormal cells quickly build up in large numbers in the bone marrow and are found in the blood.

AML is a life-threatening disease because these immature cells take the place of the normal white blood cells, reducing the patient’s ability to fight infections.

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

At the time of designation, AML affected less than 2 in 10,000 people in the European Union (EU)*. This is equivalent to a total of fewer than 101,000 people, and is below the threshold for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and knowledge of the Committee for Orphan Medicinal Products (COMP).

*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. This represents a population of 506,500,000 (Eurostat 2010).

What treatments are available?

Treatment for AML 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. At the time of designation, the main treatments for AML were chemotherapy (medicines to treat cancer) and bone marrow transplantation (a complex procedure where the bone marrow of the patient is destroyed and replaced with bone marrow from a matched donor).

The sponsor has provided sufficient information to show that (1S, 2S, 3R, 4R)-3-(5-fluoro-2-(3-methyl-4-(4-methylpiperazin-1-yl)-phenylamino)-pyrimidin-4-ylamino)-bicyclo[2.2.1]hept-5-ene-2-carboxamide benzoate might be of significant benefit for patients with AML because early studies show that it might improve the treatment 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 expected to work by blocking enzymes called ‘aurora kinases’. These enzymes play an essential role in the division of cells, by controlling the movement of chromosomes. By blocking aurora kinases, the medicine is expected to disrupt the division of cancerous cells in AML, slowing down the development of the disease.

What is the stage of development of this medicine?

The effects of this medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with this medicine that included patients with AML were ongoing.

At the time of submission, this medicine was not authorised anywhere in the EU for AML or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 6 January 2010 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.

Key facts

Product details for <p>(1S, 2S, 3R, 4R)-3-(5-Fluoro-2-(3-methyl-4-(4-methylpiperazin-1-yl)-phenylamino)-pyrimidin-4-ylamino)-bicyclo[2.2.1]hept-5-ene-2-carboxamide benzoate</p>
Active substance(1S, 2S, 3R, 4R)-3-(5-Fluoro-2-(3-methyl-4-(4-methylpiperazin-1-yl)-phenylamino)-pyrimidin-4-ylamino)-bicyclo[2.2.1]hept-5-ene-2-carboxamide benzoate
Medicine Name
Disease/conditiontreatment of acute myeloid leukaemia
Date of decision23/03/2010
Orphan decision numberEU/3/10/729

Review of designation

Sponsor’s contact details

Merck KGaA
Frankfurter Str. 250
64293 Darmstadt
Telephone: +49 61 51 72 70 0
Telefax: +49 61 51 72 20 00

Patient 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.