EU/3/13/1223 - orphan designation for treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma
Inecalcitol
OrphanHuman
Overview
On 16 January 2014, orphan designation (EU/3/13/1223) was granted by the European Commission to Hybrigenics SA, France, for inecalcitol for the treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma.
Chronic lymphocytic leukaemia (CLL) is cancer of a type of white blood cells 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 mainly affects older people and is rare in people under the age of 40 years. It 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.
At the time of designation, CLL/SLL affected approximately 3.5 in 10,000 people in the European Union (EU). This was equivalent to a total of around 179,000 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 28), Norway, Iceland and Liechtenstein. This represents a population of 512,200,000 (Eurostat 2013).
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 this medicine might be of significant benefit for patients with CLL/SLL because early clinical studies show that it might improve the outcome for patients and that it could delay the progression of the disease. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
Inecalcitol is a type of vitamin D (a 'vitamin D analogue') and attaches to the vitamin D receptor which is involved in turning genes 'on' and 'off' within cells. In CLL/SLL, inecalcitol is expected to switch 'on' the genes that suppress the division and growth of the tumour cells and to switch 'off' those genes that stimulate the division and growth of the tumour cells. This is expected to lead to a reduction in the growth and division of the cancer cells, thereby slowing the progression of the disease.
The effects of inecalcitol have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with inecalcitol in patients with CLL/SLL were ongoing.
At the time of submission, inecalcitol 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 12 December 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
- Inecalcitol
- Intended use
- Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma
- Orphan designation status
- Positive
- EU designation number
- EU/3/13/1223
- Date of designation
- Sponsor
Hybrigenics SA
3-5 impasse Reille
75014 Paris
France
Tel. +33 158 1038 00
Fax +33 158 1038 40
E-mail: inecalcitol@hybrigenics.com
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
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: