EU/3/03/141

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Orphan designation

Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in July 2014 on request of the Sponsor.

On 8 May 2003, orphan designation (EU/3/03/141) was granted by the European Commission to Bioenvision Limited, United Kingdom, for 2-chloro-9-[2-deoxy-2’-fluoro-ß-D-arabinofuranosyl]adenine for the treatment of acute myeloid leukaemia.

The sponsorship was transferred to Genzyme Europe BV, The Netherlands, in May 2008.

For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.

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 is the estimated number of patients affected by the condition?

At the time of designation, acute myeloid leukaemia affected approximately 0.7 in 10,000 people in the European Union (EU). This was equivalent to a total of around 27,000 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).


*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. At the time of designation, this represented a population of 382,800,000 (Eurostat 2003).

What treatments are 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 leukemia is chemotherapy (using drugs to kill cancer cells) and currently there exist some drugs that can be used for the treatment of this condition. 2-chloro-9-[2-deoxy-2’-fluoro-ß-D-arabinofuranosyl]adenine, also known as clofarabine, could be of potential significant benefit for the treatment of acute myeloid leukaemia. This is because it may act in a more effective way than other drugs. The assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

How is this medicine expected to work?

Clofarabine is similar to a substance called adenine. Adenine is part of the fundamental genetic material of cells (DNA and RNA). Clofarabine can block the build up of genetic material and this might kill the cancer cells.

Clofarabine received orphan drug designation in the United States in 2002 for the treatment of primary refractory or relapsed acute myelogenous leukaemia in paediatric and adult patients.

What is the stage of development of this medicine?

The effects of clofarabine were evaluated in experimental models. Clinical studies with clofarabine in patients with acute myeloid leukaemia were ongoing at the time of submission of the application for orphan designation.

Clofarabine received orphan drug designation in the United States in 2002 for the treatment of primary refractory or relapsed acute myelogenous leukaemia in paediatric and adult patients.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 19 March 2003 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>2-Chloro-9-[2-deoxy-2-fluoro-&#223;-D-arabinofuranosyl]adenine</p>
Active substance2-Chloro-9-[2-deoxy-2-fluoro-ß-D-arabinofuranosyl]adenine
Medicine Name
Disease/conditionTreatment of acute myeloid leukaemia
Date of decision08/05/2003
OutcomeWithdrawn
Orphan decision numberEU/3/03/141

Review of designation

Sponsor’s contact details

Genzyme Europe BV
Gooimeer 10
1411 DD Naarden
The Netherlands
Tel. +31 35 699 1233
Fax +31 35 699 1444
E-mail: eumedinfo@genzyme.com

Patients' organisations

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.