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Orphan designation

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in April 2014 at the end of the period of market exclusivity.

On 18 September 2001, orphan designation EU/3/01/055 was granted by the European Commission to Lipomed GmbH, Germany, for cladribine (subcutaneous use) for the treatment of indolent non-Hodgkin’s lymphoma.

Cladribine (subcutaneous use) has been authorised in the EU as Litak since 14 April 2004.

For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.

What is indolent non-Hodgkin’s lymphoma?

Non-Hodgkin's lymphomas are a type of cancer of the lymphatic system. The lymphatic system is part of the immune system: the body's natural defence against infection and disease. It is a complex system made up of organs such as bone marrow (the spongy tissue inside the large bones in the body), the thymus and the spleen, and a network of lymph nodes throughout the body that are connected by lymphatic vessels. Normally, the proliferation of lymphatic cells takes place in a controlled manner but, in non-Hodgkin’s lymphoma, this process gets out of control and the cells continue to divide, developing into a tumour. Lymphoma cells generally grow in lymph nodes. Sometimes the lymphoma cells spread from the original site to affect other lymph nodes and they may occasionally enter the bloodstream which carries them to various organs making the cancer spread. There are about 20 different types of non-Hodgkin’s lymphoma. Indolent or low-grade (slow-growing) non-Hodgkin’s lymphoma including hairy cell leukaemia, chronic lymphocytic leukaemia, grade I and II follicular lymphoma and lymphoplasmacytic lymphoma is characterised by a very slow growth of the cancer. Indolent non-Hodgkin’s lymphoma is a serious and life-threatening condition.

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

At the time of designation, indolent non-Hodgkin’s lymphoma affected 2.4 to 3.65 in 10,000 people in the European Union (EU). This was equivalent to a total of between 90,000 and 137,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased 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. At the time of designation, this represented a population of 375,500,000 (Eurostat 2000).

What treatments are available?

Currently the first treatment is chemotherapy (using drugs to kill cancer cells). Radiotherapy (using high-dose x-rays or other high-energy rays to kill cancer cells) can be useful to treat specific areas. Interferon alpha is a protein normally produced by the body during viral infections, such as flu, and is used for the treatment of certain types of non-Hodgkin’s lymphoma. There are currently several medicinal products authorised in the Community for treatment of indolent non-Hodgkin’s lymphoma. Cladribine is currently marketed within the European Union. It is generally given by slow infusion into a vein (either for two hours per day or continuously). Cladribine injected under the skin (subcutaneous use) could be of potential significant benefit for the treatment of indolent non-Hodgkin’s lymphoma. It might be as active as cladribine given through a vein. Because it is injected under the skin, it may offer a convenient way of giving cladribine and a shorter duration of treatment. These assumptions 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?

Cladribine (subcutaneous use) is purine nucleoside analogue. The purine nucleosides are part of the fundamental genetic material of cells (DNA). Cladribine (subcutaneous use) inhibits the synthesis of DNA and the DNA repair mechanism by competing with the natural purine nucleosides and thus inhibits growth of tumour cells.

What is the stage of development of this medicine?

At the time of submission of the application for orphan designation, several clinical trials in patients with indolent non-Hodgkin’s lymphoma were completed and some were ongoing.

Cladribine (subcutaneous use) was registered in Switzerland for indolent non-Hodgkin’s lymphoma at the time of submission. Orphan designation of cladribine (subcutaneous use) had not been granted in any country worldwide for non-Hodgkin’s lymphoma at the time of submission.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 18 July 2001 recommending the granting of this designation.

Update: Cladribine (subcutaneous use) (Litak) has been authorised in the EU since 14 April 2004 for treatment of hairy cell leukaemia.

Opinions on orphan medicinal products designations are based on the following cumulative 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.

Key facts

Product details for <p>Cladribine</p>
Active substanceCladribine
Medicine NameLitak
Disease/conditionTreatment of indolent non-Hodgkin’s lymphoma
Date of decision18/09/2001
Orphan decision numberEU/3/01/055

Review of designation

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in April 2014 at the end of the period of market exclusivity.

Related information

Sponsor’s contact details

Lipomed GmbH
Hegenheimer Strasse 2
D-79576 Weil am Rhein
Tel +49 7621 1693 472
Fax +49 7621 1693 474

Patients’ organisations

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.