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Orphan designation

On 9 October 2015, orphan designation (EU/3/15/1566) was granted by the European Commission to Lymphact - Lymphocyte Activation Technologies S.A., Portugal, for autologous human peripheral blood Vdelta1+ T lymphocytes activated in vitro by cytokine and monoclonal antibody treatment for the treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma.

What is chronic lymphocytic leukaemia / small lymphocytic lymphoma?

Chronic lymphocytic leukaemia (CLL) is cancer of a type of white blood cell called B lymphocytes. In this disease, the lymphocytes multiply too quickly and live for too long, so that there are too many of them circulating in the blood. The cancerous lymphocytes look normal, but they are not fully developed and do not work properly. Over time, the abnormal cells replace the normal white cells, red cells and platelets (components that help the blood to clot) in the bone marrow (the spongy tissue inside the large bones in the body).

The disease known as ‘small lymphocytic lymphoma’ (SLL) is the same disease as CLL. The name SLL is used when the cancer cells are located mainly in the lymph nodes.

CLL/SLL is the most common type of leukaemia and mainly affects older people. It is rare in people under the age of 40 years. CLL/SLL is a long-term debilitating and life-threatening disease because some patients develop severe infections.

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

At the time of designation, CLL/SLL affected approximately 4.8 in 10,000 people in the European Union (EU). This was equivalent to a total of around 246,000 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 (EU 28), Norway, Iceland and Liechtenstein. This represents a population of 512,900,000 (Eurostat 2015).

What treatments are available?

Treatment for CLL/SLL is complex and depends on a number of factors, including the extent of the disease, whether it has been treated before, and the patient’s age, symptoms and general state of health. Patients whose CLL/SLL is not causing any symptoms or is getting worse only very slowly may not need treatment. Treatment for CLL/SLL is started only if symptoms become troublesome. At the time of designation, the main treatment for CLL/SLL was chemotherapy (medicines to treat cancer).

The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with CLL/SLL because early studies in experimental models show that it might be effective at killing the cancer cells. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

This medicine contains certain T cells taken from the patient. T cells are cells of the immune system (the body’s natural defences) that kill infected or abnormal cells. To make this medicine, the T cells taken from the patient are purified to obtain certain T cells called Vdelta1+ T cells. When the medicine is injected back into the patient, Vdelta1+ T cells are expected to attach to the cancerous B lymphocytes in CLL/SLL and kill them.

What is the stage of development of this medicine?

The effects of the medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials with the medicine in patients with CLL/SLL had been started.

At the time of submission, the medicine was not authorised anywhere in the EU for CLL/SLL or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 3 September 2015 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>Autologous human peripheral blood Vdelta1+ T lymphocytes activated in vitro by cytokine and monoclonal antibody treatment</p>
Active substanceAutologous human peripheral blood Vdelta1+ T lymphocytes activated in vitro by cytokine and monoclonal antibody treatment
Medicine Name
Disease/conditionTreatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma
Date of decision09/10/2015
Orphan decision numberEU/3/15/1566

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

Lymphact - Lymphocyte Activation Technologies S.A.
Parque Tecnológico de Cantanhede, Lote 2
3060-197 Cantanhede
Tel. +351 266 092 104

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.