EU/3/03/141

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Orphan designation

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.

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 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.

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

Based on the information provided by the sponsor and previous knowledge of the Committee, acute myeloid leukaemia was considered to affect approximately 0.7 in 10,000 persons in the European Union, which, at the time of designation, corresponded to about 26,000 persons.

* Disclaimer: The number of patients affected by the condition is estimated and assessed for the purpose of the designation, for a European Community population of 377,000,000 (Eurostat 2001). This estimate is based on available information and calculations presented by the sponsor at the time of the application and may thus differ from the true number of patients affected by the condition.

How is this medicinal product expected to act?

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 medicinal product?

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.

According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 19 March 2003 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 be affecting 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 have been 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>2-chloro-9-[2-deoxy-2-fluoro-&szlig;-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
OutcomePositive
Orphan decision numberEU/3/03/141

Review of designation

Sponsor’s contact details:

Genzyme Europe BV
Gooimeer 10
1411 DD Naarden
The Netherlands
Telephone: +31 35 699 12 00
Telefax: +31 35 694 32 14
E-mail: behruz.eslami@genzyme.com