Please note that this product was withdrawn from the Community register of designated orphan medicinal products in March 2013 on request of the sponsor.
On 20 November 2001, orphan designation EU/3/01/063 was granted by the European Commission to Voisin Consulting S.A.R.L., France, for deoxyribose phosphorothioate (5’-tct-ccc-agc-gtg-cgc-cat-3’) for the treatment of chronic lymphocytic leukaemia. The sponsorship was transferred to Avensis Pharma in January 2003 and back to Voisin Consulting S.A.R.L. in July 2005.
For a list of the administrative updates to this public summary of opinion, please refer to the PDF document below.
- What is chronic lymphocytic leukaemia?
Chronic lymphocytic 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. Chronic lymphocytic leukaemia is a cancer of the white blood cells called lymphocytes. The lymphocytes multiply too quickly and live too long, so there are too many of them circulating in the blood. These leukaemic lymphocytes look normal, but they are not fully developed and do not work properly. Over a period of time these abnormal cells replace the normal white cells, red cells and platelets in the bone marrow. It is the most common type of leukaemia. Chronic lymphocytic leukaemia mainly affects older people and is rare in people under the age of 40. Chronic lymphocytic leukaemia is chronically debilitating and life-threatening due to the severe prognosis and the poor long-term survival for high-risk patients.
- What is the estimated number of patients affected by the condition?
At the time of designation, chronic lymphocytic leukaemia affected approximately 2.4 in 10,000 people in the European Union (EU). This was equivalent to a total of around 91,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This is based 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 (EU). At the time of designation, this represented a population of 378,800,000 (Eurostat 2001).
- 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. Some people with chronic lymphocytic leukaemia never have treatment if their illness is not causing any symptoms and is progressing only very slowly. Treatment is only started if and when the symptoms become
troublesome. The current main treatment for chronic lymphocytic leukemia 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. Deoxyribose phosphorothioate (5’-tct-ccc-agc-gtg-cgc-cat-3’) could be of potential significant benefit for the treatment of chronic lymphocytic leukaemia. The potential significant benefit is mainly because it might improve the long-term outcome of the patients. This 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?
So-called programmed cell death (apoptosis) is a process that is very important for the natural death of cells. A certain protein (B-cell lymphoma-associated protein 2) participates in regulating this process of cell death. In chronic lymphocytic leukaemia there is too much of this protein and the cells live longer then normal. Deoxyribose phosphorothioate (5’-tct-ccc-agc-gtg-cgc-cat-3’) might prevent the formation of this protein, decrease the amount of protein and might thereby restore normal cell death.
- What is the stage of development of this medicine?
The effects of deoxyribose phosphorothioate (5’-tct-ccc-agc-gtg-cgc-cat-3’) have been evaluated in experimental models.
At the time of submission of the application for orphan designation, several clinical trials in patients with chronic lymphocytic leukemia had been completed and some were ongoing.
Deoxyribose phosphorothioate (5’-tct-ccc-agc-gtg-cgc-cat-3’) was not marketed anywhere worldwide for chronic lymphocytic leukaemia at the time of submission.
Orphan designation of deoxyribose phosphorothioate (5’-tct-ccc-agc-gtg-cgc-cat-3’) was pending in the United States.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 7 September 2001 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.
|Name||Language||First published||Last updated|
|EU/3/01/063: Public summary of positive opinion for orphan designation of deoxyribose phosphorothioate (5’-tct-ccc-agc-gtg-cgc-cat-3’) for the treatment of chronic lymphocytic leukemia||(English only)||05/01/2005||09/04/2013|
|Active substance||Deoxyribose phosphorothioate (5'-tct-ccc-agc-gtg-cgc-cat-3')|
|Disease/condition||Treatment of chronic lymphocytic leukaemia|
|Date of decision||20/11/2001|
|Orphan decision number||EU/3/01/063|
Review of designation
Sponsor’s contact details
Voisin Consulting S.A.R.L.
3, rue des Longs Prés
Tel. +33 1 41 31 83 00
Fax +33 1 41 31 83 09
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.