EU/3/12/1058 - orphan designation for treatment of glioma
IL-12-secreting dendritic cells, loaded with autologous tumour lysate
OrphanHuman
Overview
On 8 November 2012, orphan designation (EU/3/12/1058) was granted by the European Commission to Activartis Biotech GmbH, Austria, for IL-12-secreting dendritic cells loaded with autologous tumour lysate for the treatment of glioma.
Glioma is a type of brain tumour that affects the 'glial' cells (the cells that surround and support the nerve cells). Patients with glioma can have severe symptoms, but the types of symptoms experienced depend on where the tumour develops in the brain.
Symptoms can include headaches, nausea (feeling sick), loss of appetite, vomiting, and changes in personality, mood, mental capacity and concentration. About a fifth of patients with glioma have seizures (fits) for months or years before the disease is diagnosed.
Glioma is a long-term debilitating and life-threatening disease because of the severe damage to the brain, and is associated with poor long-term survival.
At the time of designation, glioma affected approximately 1 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 51,000 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 506,300,000 (Eurostat 2011).
At the time of designation, several medicines were authorised for the treatment of glioma in the EU. Treatments for glioma included surgery, radiotherapy (treatment with radiation) and chemotherapy (medicines to treat cancer) to improve survival.
Patients also received treatments for the symptoms of glioma, including corticosteroids to reduce pressure within the skull and medicines to prevent seizures.
The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with glioma because it works in a different way to existing treatments and early studies show that it might improve the long-term outcome for these patients. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
This medicine is expected to work by activating the patient's immune system (the body's natural defences) so that it attacks and kills the cancer cells.
To prepare this medicine, immune system cells called dendritic cells are taken from the patient. The dendritic cells are then loaded with a mixture of proteins from the patient's tumour and enabled to release a substance called interleukin-12 (IL-12). When the dendritic cells are given back to the patient, the proteins from the tumour are expected to be recognised as 'foreign' by the immune system, resulting in the stimulation of an immune response against the cancer cells in the body.
The IL-12 produced by the dendritic cells is expected to enhance the immune system response against the cancer cells.
The effects of this medicine have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with this medicine in patients with glioma were ongoing.
At the time of submission, this medicine was not authorised anywhere in the EU for glioma 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 5 October 2012 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
- IL-12-secreting dendritic cells, loaded with autologous tumour lysate
- Intended use
- Treatment of glioma
- Orphan designation status
- Positive
- EU designation number
- EU/3/12/1058
- Date of designation
- Sponsor
Activartis Biotech GmbH
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: