EU/3/09/631 - orphan designation for treatment of glioma
talampanel
OrphanHuman
Overview
Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in March 2011 on request of the sponsor.
On 29 April 2009, orphan designation (EU/3/09/631) was granted by the European Commission to Teva Pharma GmbH, Germany, for talampanel 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 symptom 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 debilitating and life-threatening disease because of the severe damage to the brain that leads to poor long-term survival.
At the time of designation, glioma affected approximately 1 in 10,000 people in the European Union (EU)*. This equivalent to a total of around 50,000 people, and is below the threshold for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and 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 504,800,000 (Eurostat 2009).
At the time of designation, several medicines were authorised for the treatment of glioma in the Community. Treatments for glioma included surgery, radiotherapy (using radiation to kill the cancer cells) and chemotherapy (medicines used to kill cancer cells). Patients also received treatments for the symptoms of glioma, including corticosteroids to reduce pressure within the skull, and medicines to prevent fits.
The sponsor has provided sufficient information to show that talampanel might be of significant benefit for patients with glioma because it works in a different way to existing medicines. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
Although the cause of glioma is largely unknown, the growth of gliomas is believed to be stimulated by a neurotransmitter called glutamate. Neurotransmitters are naturally-occurring chemicals in the nervous system that allow nerve cells to communicate with each other. Glioma cells release large amounts of glutamate, which can lead to the over-activation of the receptors that glutamate normally attaches to. This results in the glioma cells stimulating their own growth and division. The over-activation of the receptors also leads to the death of the nerve cells surrounding the tumour.
Talampanel is expected to work by blocking a type of glutamate receptor on the surface of the glioma cells called AMPA receptor. This is expected to stop the signals that stimulate the glioma cells to grow and divide, slowing down the growth of the tumour and reducing the symptoms of the disease.
The effects of talampanel have been evaluated in experimental models.
At the time of submission of the application for orphan designation, a clinical trial in patients with glioma was ongoing.
At the time of submission, talampanel was not authorised anywhere in the EU for glioma or designated as 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 4 March 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 Community) 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
- talampanel
- Intended use
- Treatment of glioma
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/09/631
- Date of designation
- Sponsor
Teva Pharma GmbH
Kandelstrasse 10
79199 Kirchzarten
Germany
Telephone: +49 76 61 98 45 10
Telefax: +49 76 61 71 59
E-mail: ekkehard.baader@teva.de
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: