EU/3/08/587

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Orphan designation

On 3 December 2008, orphan designation (EU/3/08/587) was granted by the European Commission to EleosInc Limited, United Kingdom, for cenersen for the treatment chronic lymphocytic leukaemia.

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”, which mature into several different types of blood cells with 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 support blood clotting. When leukaemia develops, the bone marrow produces large numbers of abnormal blood cells. Over a period of time these abnormal cells replace the normal white cells, red cells and platelets in the bone marrow, which reduces the number of normal cells in the blood and leads to anaemia, coagulation problems (bruising, haemorrhages) and repeated infections. There are several types of leukaemias. Chronic lymphocytic leukaemia is a cancer of a type of white blood cells called B-lymphocytes. The lymphocytes multiply 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. Chronic lymphocytic leukaemia is the most common type of leukaemia; it mainly affects older people, being 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 between 1 and 3 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 and 151,000 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 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, symptoms, and the general health of the patient. Some people with B-cell chronic lymphocytic leukaemia never require treatment, if their illness is not causing any symptoms and is progressing slowly. Treatment is often started only if and when the symptoms become troublesome.

Current main treatment of chronic lymphocytic 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.

How is this medicine expected to work?

The cells have natural mechanisms that induce their death when some alterations occur, for instance when their genetic material is damaged. One of the mechanisms of cell death is controlled by a gene called p53. This gene makes cells to undergo either repair of the damages or cell death. In cancer cells, due to a complicated association of alterations, p53 activation tends to result in repair even though this is not complete and finally leads to production of more damaged cells and therefore multiplication of the tumour. The product is able to block both p53 and mutated p53; by doing this it blocks p53 mediated cell repair cancer so cells can undergo cell death via an alternative pathway.

What is the stage of development of this medicine?

At the time of submission of the application for orphan designation, clinical trials in patients with chronic lymphocytic leukaemia were ongoing.

At the time of submission, cenersen was not authorised anywhere in the world for chronic lymphocytic leukaemia. Orphan designation of cenersen had been granted in the United States for treatment of chronic lymphocytic leukaemia.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 8 October 2008 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;
  • 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>Cenersen</p>
Active substanceCenersen
Medicine Name
Disease/conditionTreatment of chronic lymphocytic leukaemia
Date of decision02/12/2008
OutcomePositive
Orphan decision numberEU/3/08/587

Review of designation

Sponsor’s contact details:

EleosInc Limited
21 Wilson Street
London EC2M 2TD
United Kingdom
Telephone: +44 20 7588 08 00
Telefax: +44 20 7588 0555
E-mail: Dreardan@EleosInc.com

Patients’ associations contact points:

Leukaemia CARE
One Birch Court
Blackpole East
Worcester
Worcestershire WR3 8SG
United Kingdom
Telephone: +44 1905 755977 / +44 845 521 3456
24 hour CARE line 0800 169 6680 (UK residents only)
Telefax: +44 1905 755166
Email: info@leukaemiacare.org.uk

Ligue Nationale Contre le Cancer
14 Rue Corvisart
75013 Paris
France
Telephone: +33 1 53 55 24 00
Telefax: +33 1 43 36 91 10
E-mail: ligue@ligue-cancer.net

Deutsche Krebshilfe e. V.
Buschstr. 32
53113 Bonn
Germany
Telephone: +49 2 287 29 900
Telefax: +49 2 287 29 90 11
E-mail: deutsche@krebshilfe.de