EU/3/08/562

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Orphan designation

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.

What is 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.

What is the estimated number of patients affected by the condition?

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).

What treatments are available?

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.

How is this medicine expected to work?

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.

What is the stage of development of this medicine?

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.

Opinions on orphan medicinal product designations are based on the following three criteria:
  • 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

Product details for <p>Topotecan hydrochloride (liposomal)</p>
Active substanceTopotecan hydrochloride (liposomal)
Medicine Name
Disease/conditionTreatment of glioma
Date of decision05/09/2008
OutcomePositive
Orphan decision numberEU/3/08/562

Review of designation

Sponsor’s contact details:

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

Patients’ associations contact points:

Ligue Nationale Contre le Cancer
14 Rue Corvisart
75013 Paris
France
Telephone: +33 1 53 55 24 00
Telefax: +33 1 43 36 91 10
E-mail: ligue@ligue-cancer.net

Deutsche Krebshilfe e. V.
Buschstr. 32
53113 Bonn
Germany
Telephone: +49 2 287 29 900
Telefax: +49 2 287 29 90 11
E-mail: deutsche@krebshilfe.de

Macmillan Cancer Support (merged with CancerBACUP)
3 Bath Place
Rivington Street
London
EC2A 3JR
United Kingdom
Telephone: +44 20 76 96 90 03
Switchboard open during office hours, Mon–Fri, 9am–Noon and 2pm–4.45pm
Telefax: +44 20 76 96 90 02