EU/3/13/1229 - orphan designation for treatment of malignant mesothelioma
autologous dendritic cells pulsed with allogeneic tumour cell lysate
OrphanHuman
Overview
On 16 January 2014, orphan designation (EU/3/13/1229) was granted by the European Commission to Amphera BV, the Netherlands, for autologous dendritic cells pulsed with allogeneic tumour cell lysate for the treatment of malignant mesothelioma.
For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.
Malignant mesothelioma is a cancer that affects the mesothelial cells (found on the inner linings of the organs), mainly in the pleura (the lining of the lungs) and in the peritoneum (the lining of the abdominal cavity). It is usually caused by exposure to asbestos. Mesothelioma of the pleura causes difficulty breathing and chest pain, and mesothelioma of the peritoneum causes ascites (a build-up of fluid in the abdomen) and abdominal pain. Because the symptoms are not specific, the cancer is often detected at a late stage.
Malignant mesothelioma is life-threatening because it may lead to bowel obstruction, heart or breathing problems and lung infections. Patients have very poor survival, only living for a year, on average, after diagnosis.
At the time of designation, malignant mesothelioma affected approximately 0.3 in 10,000 people in the European Union (EU). This was equivalent to a total of around 15,000 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. At the time of designation, this represented a population of 512,900,000 (Eurostat 2014).
At the time of designation, the main treatment for malignant mesothelioma was surgery followed by chemotherapy (medicines to treat cancer) or radiotherapy (treatment with radiation). If the disease was too advanced for surgery, chemotherapy alone was used. Only one medicine, pemetrexed, was specifically authorised in the EU for the treatment of malignant pleural mesothelioma.
The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with malignant mesothelioma because it works in a different way to current treatments and early preclinical studies suggested it might improve survival of patients with the condition. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
The medicine works by activating the patient's immune system (the body's natural defences) so that it attacks and kills the cancer cells. The medicine is prepared from the patient's own (autologous) immune cells called dendritic cells. These cells are mixed in the laboratory with components of five different types of malignant mesothelioma cells. When the dendritic cells are injected back into the patient, it is expected that they will help the immune system recognise similar components in the patient's own malignant mesothelioma cells as 'foreign' and stimulate an immune response against them, helping to slow down or stop the progression of the disease.
The effects of the medicinal product have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with the medicinal product in patients with malignant mesothelioma were ongoing.
At the time of submission, the medicinal product was not authorised anywhere in the EU for malignant mesothelioma 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
- autologous dendritic cells pulsed with allogeneic tumour cell lysate
- Intended use
- Treatment of malignant mesothelioma
- Orphan designation status
- Positive
- EU designation number
- EU/3/13/1229
- Date of designation
- Sponsor
Amphera BV
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: