EU/3/13/1186 - orphan designation for treatment of extranodal marginal-zone lymphoma of mucosa-associated lymphoid tissue (MALT lymphoma)
Idelalisib
OrphanHuman
Overview
Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in October 2013 on request of the sponsor.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/3/13/1186) was granted by the European Commission to Gilead Sciences International Ltd, United Kingdom, for idelalisib for the treatment of extranodal marginal-zone lymphoma of mucosa-associated lymphoid tissue (MALT lymphoma).
For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.
Extranodal marginal-zone lymphoma of mucosa-associated lymphoid tissue (MALT lymphoma) is a cancer of a type of white blood cell called B lymphocytes or B cells. The abnormal B cells multiply too quickly and live for too long, so there are too many of them. In MALT lymphoma the abnormal B cells affect various organs, particularly the gut, but also thyroid gland, lung, breast, eye and skin. Patients usually have fever, weight loss, tiredness and night sweats.
MALT lymphoma is a life-threatening and long-term debilitating disease due to gut problems and the risks of bone marrow damage and of the disease transforming to a more aggressive form of lymphoma.
At the time of designation, MALT lymphoma affected approximately 0.4 in 10,000 people in the European Union (EU). This was equivalent to a total of around 20,000 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).
At the time of designation, the main treatments for MALT lymphoma used in the EU included immunotherapy (using the body's own immune system to kill cancer cells) with the medicine rituximab, chemotherapy (anticancer medicines), radiotherapy (treatment with radiation) and surgery to remove affected lymph nodes. In some patients, MALT lymphoma affecting the stomach is associated with infection by the bacterium Helicobacter pylori, and treatment with antibiotics was used to resolve the infection.
The sponsor has provided sufficient information to show that idelalisib might be of significant benefit for patients with MALT lymphoma because early studies in patients with marginal-zone lymphoma show that it might improve the outcome of patients whose disease was resistant to or had come back after existing treatments. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
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 plays an important role in the growth, migration and survival of white blood cells. In patients with MALT lymphoma, PI3K-delta is active in the abnormal B cells, stimulating their growth and survival. By blocking its effects, the medicine is expected to reduce the growth and survival of the abnormal B cells.
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 MALT lymphoma were ongoing.
At the time of submission, idelalisib was not authorised anywhere in the EU for MALT lymphoma. Orphan designation of idelalisib had been granted in the EU for lymphoplasmacytic lymphoma and follicular lymphoma, and 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 11 July 2013 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.
Key facts
- Active substance
- Idelalisib
- Intended use
- Treatment of extranodal marginal-zone lymphoma of mucosa-associated lymphoid tissue (MALT lymphoma)
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/13/1186
- Date of designation
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.
EMA list of opinions on orphan medicinal product designation
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
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.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: