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Orphan designation

On 10 October 2012, orphan designation (EU/3/12/1055) was granted by the European Commission to Topotarget A/S, Denmark, for belinostat for the treatment of peripheral T-cell lymphoma (nodal, other extranodal and leukaemic / disseminated).

The sponsorship was transferred to Onxeo DK Filial af Onxeo S.A., Frankrig, Denmark, in June 2015.

What is peripheral T-cell lymphoma?

Peripheral T-cell lymphoma is a cancer of the lymphatic system, a network of vessels that transport fluid from tissues through the lymph nodes and into the bloodstream. In peripheral T-cell lymphoma there is uncontrolled growth of T lymphocytes (T cells), a type of white blood cell found in the lymphatic system. Different types of peripheral T-cell lymphoma have been identified and categorised as nodal, other extranodal and leukaemic / disseminated.

The symptoms of the disease vary according to the type of lymphoma, but the first sign is usually a lump in the neck, under the arm or in the groin area, which is caused by an enlarged lymph node. The lymphoma may also affect other organs in the body such as the bone marrow, liver and the skin.

Peripheral T-cell lymphoma is a long-term debilitating and life-threatening condition because in most cases the disease does not respond well to therapy and comes back within one year after initial treatment and is associated with poor overall survival.

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

At the time of designation, peripheral T-cell lymphoma affected less than 1 in 10,000 people in the European Union (EU)*. This is equivalent to a total of fewer than 51,000 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 27), Norway, Iceland and Liechtenstein. This represents a population of 506,300,000 (Eurostat 2011).

How is this medicine expected to work?

Belinostat is an ‘HDAC inhibitor’ medicine. This means that it blocks enzymes called histone deacetylases (HDACs), which are involved in turning genes ‘on’ and ‘off’ within cells. By blocking HDAC enzymes, belinostat is expected to ‘switch on’ the genes that suppress the division and growth of the tumour cells in peripheral T-cell lymphoma. This is expected to lead to a reduction in the growth and division of the cancer cells.

What is the stage of development of this medicine?

The effects of belinostat have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with the medicine in patients with peripheral T-cell lymphoma were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for peripheral T-cell lymphoma. Orphan designation had been granted in the United States for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 5 September 2012 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.

Key facts

Product details for <p align="left">Belinostat</p>
Active substanceBelinostat
Medicine Name
Disease/conditionTreatment of peripheral T-cell lymphoma (nodal, other extranodal and leukaemic / disseminated)
Date of decision10/10/2012
Orphan decision numberEU/3/12/1055

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor’s contact details

Onxeo DK, Filial af Onxeo S.A., Frankrig
c/o Symbion
Fruebjergvej 3
2100 Copenhagen Ø
Tel. +45 3917 9494
Fax +45 3917 9492

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.