EU/3/06/428

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Orphan designation

On 29 January 2007, orphan designation (EU/3/06/428) was granted by the European Commission to Napp Pharmaceuticals Research Limited, United Kingdom, for forodesine hydrochloride for the treatment of cutaneous T cell lymphoma.

The name of the sponsor changed to Mundipharma Research Limited in 2007.

What is cutaneous T cell lymphoma?

Cutaneous T cell lymphoma is a type of cancer of the lymphatic system. The lymphatic system is part of the body’s immune system and helps fighting infections. It is a complex system made up of organs such as the bone marrow, the thymus (a gland behind the breast bone), the spleen (an organ in the abdomen, near the stomach), and the lymph nodes (or lymph glands, located throughout the body), which are connected by a network of tiny lymphatic vessels; in addition, there are isolated lymphatic cells in most tissues of the body, including the skin. One of the main cells that make up the lymphatic tissue is the lymphocyte, which belongs to the group of white blood cells. There are two main types of lymphocytes: B-lymphocytes (B cells) and T-lymphocytes (T cells). Cutaneous T-cell lymphoma is characterised by uncontrolled growth of the T-lymphocytes, mainly affecting the skin. This condition most often occurs in people aged between 40 and 60. Although in many cases the condition is chronic and survival for more than 10-20 years is common, cutaneous T-cell lymphoma can be a serious and life-threatening condition.

What are the methods of treatment available?

Current treatment for cutaneous T-cell lymphoma can be divided into local treatments and systemic (given to the whole body) treatments. Local treatments include medicines applied to the skin, therapies using light of a particular wavelength (ultraviolet light) and x-rays. Systemic treatments included medicines such as glucocorticoids (a group of medicines that are similar to cortisone), cytotoxic agents (medicines that kill cells), interferon-alfa (a compound that can help the immune system to fight against the cancer cells) and photopheresis (blood is temporarily removed from the body and exposed to ultraviolet light, after being treated with a sensitizing compound). Several products were authorised for the treatment of cutaneous T cell lymphoma within the Community at the time of submission of the application for orphan drug designation. Forodesine might be of potential significant benefit for the treatment of cutaneous T-cell lymphoma because it is expected to act in a different way than other available medicines. This assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status of forodesine.

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

According to the information provided by the sponsor, cutaneous T cell lymphoma was considered to affect about 66,000 persons in the European Union.

* 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 25), Norway, Iceland and Lichtenstein. This represents a population of 459,700,000 (Eurostat 2004). 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?

Forodesine hydrochloride is a substance acting specifically on an enzyme called purine nucleoside phosphorylase (PNP). Enzymes are proteins produced by the cells of the body; they speed up the conversion of certain substances into other substances. Forodesine is an inhibitor of PNP. Reducing the activity of this enzyme may cause the accumulation of a substance, called dGTP, which is toxic to these cells if present in high concentrations. Human lymphocytes, particularly malignant T- and B-cells may be susceptible to forodesine, because they have a relatively high activity of another enzyme, dCK, which actually generates dGTP, and a low activity of an opposing enzyme (5’ nucleotidase), which instead would prevent dGTP accumulation. Since cutaneous T cell lymphoma is caused by the uncontrolled growth of the malignant T-cells lymphocytes, forodesine might help in slowing down or stopping this uncontrolled cell growth.

What is the stage of development of this medicinal product?

The effects of forodesine are being evaluated in preliminary clinical studies in humans; one study has been completed and one is ongoing.
Forodesine hydrochloride was not authorised anywhere worldwide for the treatment of cutaneous T cell lymphoma, at the time of submission of the request for orphan drug designation.

According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 6 December 2006 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>Forodesine hydrochloride</p>
Active substanceForodesine hydrochloride
Medicine Name
Disease/conditionTreatment of cutaneous T cell lymphoma
Date of decision28/01/2007
OutcomePositive
Orphan decision numberEU/3/06/428

Review of designation

Sponsor’s contact details:

Mundipharma Research Limited
Cambridge Science Park
Milton Road
Cambridge
Cambridgeshire CB4 0WG
United Kingdom
Telephone: +44 1223 424 900
Telefax: +44 1223 426 054
E-mail: medicalinformationuk@napp.co.uk

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