EU/3/07/476

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Orphan designation

On 14 September 2007, orphan designation (EU/3/07/476) was granted by the European Commission to Clavis Pharma ASA, Norway, for 5'-O-(trans-9"-octadecenoyl)-1-ß-D-arabinofuranosyl cytosine 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”, which 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 should develop into a type of 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; they also accumulate in the bone marrow where they take the place of the other types of normal blood cells, causing anaemia, easy bruising, and frequent infections. Myeloid 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. Satisfactory argumentation has been submitted by the sponsor to justify the assumption that 5'-O-(trans-9"-octadecenoyl)-1-ß-D-arabinofuranosyl cytosine might be of potential significant benefit for the treatment of acute myeloid leukaemia because it might possibly be used when other treatments have failed. This 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 2 in 10,000 persons in the European Union, which, at the time of designation, corresponded to about 100,000 persons.

*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 Lichtenstein. This represents a population of 498,000,000 (Eurostat 2006). 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?

5'-O-(trans-9"-octadecenoyl)-1-ß-D-arabinofuranosyl cytosine is a product similar to cytidine, which in turn is a building block of the fundamental genetic material of cells (DNA and RNA). 5'-O-(trans-9"-octadecenoyl)-1-ß-D-arabinofuranosyl cytosine blocks the synthesis of DNA and RNA. By doing this the drug stops the production of genetic material necessary for cell growth and division. Thus, the drug inhibits the growth cells and specially tumour cells which grow and duplicate very actively.

What is the stage of development of this medicinal product?

The effects of 5'-O-(trans-9"-octadecenoyl)-1-ß-D-arabinofuranosyl cytosine 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.

5'-O-(trans-9"-octadecenoyl)-1-ß-D-arabinofuranosyl cytosine was not authorised 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 25 July 2007 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>5'-O-(trans-9"-octadecenoyl)-1-&szlig;-D-arabinofuranosyl cytosine</p>
Active substance5'-O-(trans-9"-octadecenoyl)-1-ß-D-arabinofuranosyl cytosine
Medicine Name
Disease/conditionTreatment of acute myeloid leukaemia
Date of decision14/09/2007
OutcomePositive
Orphan decision numberEU/3/07/476

Review of designation

Sponsor’s contact details:

Clavis Pharma ASA
Parkveien 53B
0256 Oslo
Norway
Telephone: + 47 24 11 09 50
Telefax: + 47 24 11 09 51
E-mail: bo.nilsson@clavispharma.com

Patients’ associations contact points:

Leukaemia Care
2 Shrubbery Avenue
Worcester
WR1 1QH
United Kingdom
0800 169 6680 (freephone for UK residents)
Telephone: +44 19 05 33 00 03 / + 44 84 57 67 32 03
Telefax: +44 19 05 33 00 90
E-mail: enquiries@leukaemiaCARE.org.uk

DLH : Deutsche Leukämie- und Lymphom-Hilfe e.V.
Thomas-Mann-Straße 40
53111 Bonn
Germany
Telephone: +49 22 83 90 440
Telefax: +49 22 83 90 44 22
E-mail: info@leukaemie-hilfe.de

Associazione Italiana contro le Leucemie-linfomi e mieloma ONLUS
Via Ravenna 34
00161 Roma
Italy
Telephone: +39 06 44 03 763
Telefax: +39 06 44 04 226
E-mail: ail@ail.it