EU/3/13/1160

  • Email
  • Help

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Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in October 2013 on request of the sponsor.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 17 July 2013, orphan designation (EU/3/13/1160) was granted by the European Commission to Gilead Sciences International Ltd, United Kingdom, for idelalisib for the treatment of lymphoplasmacytic lymphoma.

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

What is lymphoplasmacytic lymphoma?

Lymphoplasmacytic lymphoma is a cancer of a type of white blood cell called B lymphocytes or B cells. In lymphoplasmacytic lymphoma, the B cells multiply too quickly and live for too long, so there are too many of them in places like the bone marrow, lymph nodes or spleen. The first signs of the disease are usually weakness and tiredness. In many patients with lymphoplasmacytic lymphoma, the abnormal B cells produce too much of a type of blood protein called immunoglobulin-type-M paraprotein (IgM paraprotein), which makes the blood too viscous (thick) and can lead to symptoms such as eye problems, heart failure, haemolytic anaemia (destruction of red blood cells) and effects on the nervous system.

Lymphoplasmacytic lymphoma is a life-threatening and long-term debilitating disease due to damage to the bone marrow and the effects of IgM paraprotein.

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

At the time of designation, lymphoplasmacytic lymphoma affected approximately 0.06 in 10,000 people in the European Union (EU). This was equivalent to a total of around 3,000 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?

At the time of designation, the main treatments for diseases such as lymphoplasmacytic lymphoma available in the EU included immunotherapy (medicines that stimulate the body’s own immune system to kill the cancer cells), and combinations of immunotherapy with chemotherapy (anticancer medicines intended to kill the cancer cells). Medicines designed to attach to the cancer cells and kill them with radiation (radioimmunotherapy) were also sometimes used.

The sponsor has provided sufficient information to show that idelalisib might be of significant benefit for patients with lymphoplasmacytic lymphoma, because early studies in patients with lymphoplasmacytic lymphoma that was resistant to or had come back after existing treatments showed that some of these patients responded to idelalisib. 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 which is a member of a family of enzymes called phosphoinositide-3-kinases (PI3K) that play an important role in the growth, migration and survival of white blood cells. PI3K-delta is active in the abnormal B cells of patients with lymphoplasmacytic lymphoma, stimulating their growth and survival. By blocking its effects, the medicine is expected to reduce the growth and survival of the abnormal B cells.

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 lymphoplasmacytic lymphoma were ongoing.

At the time of submission, idelalisib was not authorised anywhere in the EU for lymphoplasmacytic lymphoma or designated as an orphan medicinal product elsewhere for this condition. Orphan designation of idelalisib had been granted in the United States for chronic lymphocytic leukaemia.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 13 June 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.

Name Language First published Last updated
EU/3/13/1160: Public summary of opinion on orphan designation: Idelalisib for the treatment of lymphoplasmacytic lymphoma (English only) 22/08/2013 18/11/2013

Key facts

Product details for <p>Idelalisib</p>
Active substanceIdelalisib
Medicine Name
Disease/conditionTreatment of lymphoplasmacytic lymphoma
Date of decision17/07/2013
OutcomeWithdrawn
Orphan decision numberEU/3/13/1160

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
Abington
Cambridge
CB21 6GT
United Kingdom
Tel. +44 (0)1223 897300
Fax +44 (0)1223 897284
E-mail: regulatory.orphan@gilead.com

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.