EU/3/09/721 - orphan designation for treatment of Hodgkin's lymphoma
Panobinostat
OrphanHuman
Overview
Please note that this product was withdrawn from the Community register of designated orphan medicinal products in April 2012 on request of the sponsor.
On 2 February 2010, orphan designation (EU/3/09/721) was granted by the European Commission to Novartis Europharm Limited, United Kingdom, for panobinostat for the treatment of Hodgkin's lymphoma.
For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.
Hodgkin's lymphoma is a type of cancer of the lymphatic system, a network of vessels that transport lymph from tissues through the lymph nodes and into the bloodstream. Because lymph nodes are found throughout the body, the cancer can begin in almost any part of the body. In Hodgkin's lymphoma, white blood cells in the lymphatic system multiply too quickly and live for too long. These cancer cells can spread through the lymphatic system to other lymph nodes or through the bloodstream to other organs such as the spleen, where they can form new tumours.
Many people with Hodgkin's lymphoma can be cured if the disease is found and treated early. However, despite the available treatments, Hodgkin's lymphoma remains a serious and life-threatening disease, mainly because it leads to poor survival in patients whose disease does not respond to treatment or has come back after previous treatment.
At the time of designation, Hodgkin's lymphoma affected between 1 and 4 in 10,000 people in the European Union (EU). This was equivalent to a total of between 51,000 and 203,000 people*, and is below the threshold 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 27), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 506,300,000 (Eurostat 2010).
At the time of designation, several medicines were authorised for the treatment of Hodgkin's lymphoma in the EU.
The main treatments for Hodgkin's lymphoma included chemotherapy (medicines to treat cancer) and radiotherapy (treatment with radiation). Autologous bone marrow transplantation was also used when the disease had not responded to treatment or had come back after treatment. This is a complex procedure where the bone marrow of the patient is destroyed and replaced with healthy bone marrow previously obtained from the same patient.
The sponsor has provided sufficient information to show that panobinostat might be of significant benefit for patients with Hodgkin's lymphoma because early studies indicate that it might improve the treatment of patients with this condition, particularly patients whose disease 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.
Panobinostat is expected to work by blocking the activity of proteins called histone deacetylases, which are involved in turning genes 'on' and 'off' within cells. In Hodgkin's lymphoma, panobinostat is expected to keep the genes that suppress the division and growth of the tumour cells switched 'on'. This is expected to lead to a reduction in the growth and division of the lymphoma cells.
The effects of panobinostat have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with the designated product in patients with Hodgkin's lymphoma were ongoing.
At the time of submission, panobinostat was not authorised anywhere in the EU for Hodgkin's lymphoma. Orphan designation of panobinostat had been granted in the United States of America for Hodgkin's lymphoma.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 3 December 2009 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 European Union) 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
- Panobinostat
- Intended use
- Treatment of Hodgkin's lymphoma
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/09/721
- Date of designation
- Sponsor
Novartis Europharm Limited
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: