EU/3/12/1055: Orphan designation for the treatment of peripheral T-cell lymphoma (nodal, other extranodal and leukaemic/disseminated)

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.

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).

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.

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.

• 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.