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Orphan designation

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in October 2013 on request of the sponsor.

On 5 August 2013, orphan designation (EU/3/13/1173) was granted by the European Commission to Gilead Sciences International Ltd, United Kingdom, for idelalisib for the treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma.

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

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 a period of 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, where blood cells are produced). The disease known as ‘small lymphocytic lymphoma’ (SLL) is essentially the same disease as CLL. The name SLL is normally 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 it involves the weakening of the immune system (the body’s natural defences), which can leave patients vulnerable to severe infections.

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

At the time of designation, CLL / SLL affected less than 3.5 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 178,000 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 509,000,000 (Eurostat 2013).

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 only getting worse very slowly may not need treatment. Treatment for CLL / SLL is only started 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 idelalisib might be of significant benefit for patients with CLL / SLL because early studies show that it might improve the outcome of patients whose disease does not respond to or has come back after previous treatment. 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?

Idelalisib blocks the effects of an enzyme called PI3K-delta, a member of a family of enzymes called phosphoinositide-3 kinases (PI3K), which play an important role in the growth, migration and survival of white blood cells. In patients with CLL / SLL, PI3K-delta is active in the abnormal B lymphocytes, stimulating their growth and survival. By blocking its effects, the medicine is expected to reduce the growth and survival of the abnormal B lymphocytes.

What is the stage of development of this medicine?

The effects of idelalisib have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with idelalisib in patients with CLL / SLL were ongoing.

At the time of submission, idelalisib was not authorised anywhere in the EU for CLL / SLL. Orphan designation of idelalisib had been granted in the United States for CLL and in the EU for lymphoplasmacytic lymphoma and follicular lymphoma.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 11 July 2013 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>Idelalisib</p>
Active substanceIdelalisib
Medicine Name
Disease/conditionTreatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma
Date of decision05/08/2013
Orphan decision numberEU/3/13/1173

Review of designation

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in October 2013 on request of the sponsor, before a marketing authorisation had been granted.

Sponsor’s contact details

Gilead Sciences International Limited
Flowers Building
Granta Park
CB21 6GT
United Kingdom
Tel. +44 (0)1223 897300
Fax +44 (0)1223 897284

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.