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Orphan designation

On 10 July 2008, orphan designation (EU/3/08/558) was granted by the European Commission to Cyclacel Limited, United Kingdom, for sapacitabine for the treatment of acute myeloid leukaemia.

What is acute myeloid leukaemia?

Acute myeloid leukaemia (AML) is a cancer of the white blood cells. In this disease, the bone marrow 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 life-threatening because these immature cells take the place of the normal white blood cells. As a result, the patient’s ability to fight diseases is reduced.

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

At the time of designation acute myeloid leukaemia affected 1 in 10,000 people in the European Union (EU)*. This is based on the information provided by the sponsor and knowledge of the Committee for Orphan Medicinal Products (COMP). This is below the threshold for orphan designation which is 5 in 10,000. This is equivalent to a total of around 50,000 people.

* 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 27), Norway, Iceland and Liechtenstein. This represents a population of 502,282,000 (Eurostat 2008).

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. The primary treatment for AML is chemotherapy (using medicines to kill cancer cells).

Satisfactory argumentation has been submitted by the sponsor to justify the assumption that sapacitabine might be of potential significant benefit for the treatment of acute myeloid leukaemia because it might improve the long-term outcome of the patients and because it is planned to be administered as capsules. These assumptions will need to be confirmed at the time of marketing authorisation, to maintain the orphan status.

How is this medicine expected to work?

Sapacitabine is expected to kill dividing cells. Sapacitabine it is a nucleoside analogue, which means that it is similar to a nucleoside, a chemical that forms part of the DNA (the fundamental genetic material in cells). In the body, sapacitabine is expected to interfere with an enzyme called DNA polymerase, which is involved in the formation of DNA. This is expected to slow down the production of DNA and the growth of the cancerous cells.

What is the stage of development of this medicine?

The effects of sapacitabine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials in patients with acute myeloid leukaemia had been started.

At the time of submission, sapacitabine was not authorised anywhere in the world for AML or designated as orphan medicinal product elsewhere for this condition.According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted a positive opinion on 14 May 2008, recommending the granting of the above-mentioned 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;
  • and either the rarity of the condition (affecting not more than five in 10,000 people in the Community) or the 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 the quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.

Key facts

Product details for <p>Sapacitabine</p>
Active substanceSapacitabine
Medicine Name
Disease/conditionTreatment of acute myeloid leukemia
Date of decision10/07/2008
Orphan decision numberEU/3/08/558

Review of designation

Sponsor’s contact details:

Cyclacel Limited
Dundee Technopole
James Lindsay Place
Dundee, DD1 5JJ
United Kingdom
Telephone: + 44 1382 20 60 62
Telefax: + 44 1382 20 60 67

Patients’ associations contact points:

Ligue Nationale Contre le Cancer
14 Rue Corvisart
75013 Paris
Telephone: +33 1 53 55 24 00
Telefax: +33 1 43 36 91 10

Deutsche Krebshilfe e. V.
Buschstr. 32
53113 Bonn
Telephone: +49 2 287 29 900
Telefax: +49 2 287 29 90 11

Macmillan Cancer Support (merged with CancerBACUP)
3 Bath Place
Rivington Street
United Kingdom
Telephone: +44 20 7696 9003
Switchboard open during office hours, Mon–Fri, 9am–Noon and 2pm–4.45pm
Telefax: +44 20 7696 9002