EU/3/08/562 - orphan designation for treatment of glioma
topotecan hydrochloride
Orphan
Human
Overview
On 5 September 2008, orphan designation (EU/3/08/562) was granted by the European Commission to Dr Matthias Luz, Germany, for topotecan hydrochloride (liposomal) for the treatment of glioma.
Glioma is a type of brain cancer that starts in the glial cells (the cells that support the nerve cells). Patients with glioma can have severe symptoms affecting the nervous system, but the types of symptom depend on where the tumour develops in the brain. Glioma is life-threatening.
At the time of designation glioma affected approximately 1 in 10,000 people in the European Union (EU) *. This is based on the information provided by the sponsor and knowledge of the Committee for Orphan Medicinal Products (COMP). This is below the threshold for orphan designation which is 5 in 10,000. This is equivalent to a total of around 50,000 people.
*Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed based on data from the European Union (EU 27), Norway, Iceland and Liechtenstein. This represents a population of 502,282,000 (Eurostat 2008).
Treatment for glioma includes surgery, radiotherapy (using radiation to kill the cancer cells) and chemotherapy (medicines used to kill cancer cells). Patients may also receive treatments for the symptoms of glioma, including corticosteroids to reduce pressure within the skull, and medicines to prevent seizures. At the time of submission of the application for orphan designation, several medicines were authorised for the treatment of glioma in the Community.
Satisfactory argumentation has been submitted by the sponsor to justify the assumption that topotecan hydrochloride (liposomal) might be of potential significant benefit for the treatment of glioma because it has a new delivery method. This assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.
Topotecan is a medicine that is already used in chemotherapy, although it has not been authorised for the treatment of glioma. Topotecan belongs to the group 'topoisomerase inhibitors'. It blocks an enzyme called topoisomerase I, which is involved in the division of DNA (the genetic material). When the enzyme is blocked, the DNA strands break. This prevents the cancer cells from dividing and they eventually die. In this medicine, topotecan is included in small fatty particles called 'liposomes'. This is expected to improve the way the medicine works compared with the conventional form of the medicine, since it can be injected directly into the brain, concentrating the medicine at the site of the tumour and reducing side effects by reducing the amount of the medicine in the blood.
The effects of topotecan hydrochloride (liposomal) have been evaluated in experimental models.
At the time of submission of the application for orphan designation, no clinical trials in patients with glioma had been initiated.
Topotecan hydrochloride (liposomal) was not authorised anywhere worldwide for the treatment of glioma or designated as orphan medicinal product elsewhere for this condition, at the time of submission.
According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted a positive opinion on 11 June 2008 recommending the granting of the above-mentioned designation.
- the seriousness of the condition;
- the existence of alternative methods of diagnosis, prevention or treatment;
- and either the rarity of the condition (affecting not more than five in 10,000 people in the Community) or the 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 the quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.
Key facts
- Active substance
- topotecan hydrochloride
- Intended use
- Treatment of glioma
- Orphan designation status
- Positive
- EU designation number
- EU/3/08/562
- Date of designation
- Sponsor
Dr Matthias Luz
Brunhildestr. 46
68199 Mannheim
Germany
Telephone: + 49 621 819 19 39
Telefax: + 49 621 819 19 49
E-mail: matthiasluz@med-genesis.com
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: