This medicine is now known as axicabtagene ciloleucel.
On 11 November 2015, orphan designation (EU/3/15/1572) was granted by the European Commission to Kite Pharma UK, Ltd, United Kingdom, for autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor for the treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma.
The sponsorship was transferred to Kite Pharma EU B.V., Netherlands, in April 2017. The sponsor’s address was updated in September 2021.
Please note that this product was withdrawn from the Union Register of orphan medicinal products in December 2021 on request of the Sponsor.
Chronic lymphocytic leukaemia (CLL) is cancer of a type of white blood cell called B lymphocytes or B cells. In this disease, the B cells multiply too quickly and live for too long, so that there are too many of them circulating in the blood. The cancerous B cells look normal, but they are not fully developed and do not work properly. Over time, the cancer cells replace the normal white cells, red cells and platelets 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 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.
At the time of designation, CLL/SLL affected approximately 4.9 in 10,000 people in the European Union (EU). This was equivalent to a total of around 251,000 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).
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 showed that patients whose disease had come back after previous treatment or did not respond to previous treatment responded to treatment with this medicine. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
The abnormal B cells in patients with CLL/SLL produce a protein on their surface called CD19.
This medicine is made up of immune cells (called T cells) which are taken from the patient and modified in the laboratory with a virus that carries a gene into the T cells so that they can recognise and attach to CD19. The modified T cells are then given back to the patient, where they are expected to attach to CD19 on the cancer cells and kill them. These T cells are also expected to activate other T cells from the patient to act against the cancer cells.
The type of virus used in this medicine ('retrovirus') is modified in order not to cause disease in humans.
The effects of this medicine have been evaluated in experimental models.
At the time of submission of the application for orphan designation, a clinical trial with the medicine including patients with CLL/SLL was ongoing.
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 8 October 2015 recommending the granting of this designation.
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.
Kite Pharma EU B.V.
Tufsteen 1
2132 NT Hoofddorp
Noord-Holland
The Netherlands
Tel. +31 2023 52630
E-mail: regulatory@kitepharma.com
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: