EU/3/07/507

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Orphan designation

On 29 November 2007, orphan designation (EU/3/07/507) was granted by the European Commission to CellMed Agf, Germany, for doxorubicin hydrochloride (drug eluting beads) for the treatment of glioma.

The sponsorship was transferred to Biocompatibles UK Limited, United Kingdom, in October 2012.

What is glioma?

Tumours that begin in brain tissue are known as primary brain tumours. Primary brain tumours are named after the type of tissue from which they develop. The most common brain tumours are gliomas, which begin in the glial (supportive) tissue.
Due to their location, gliomas represent a potentially debilitating and life-threatening condition. Patients affected by gliomas can suffer from severe symptoms of the nervous system, depending on where in the brain the tumour develops. 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 was equivalent to a total of around 50,000 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. At the time of designation, this represented a population of 500,300,000 (Eurostat 2007).

What treatments are available?

Treatment for glioma depends on a number of factors, and may include surgery, radiotherapy or chemotherapy as well as symptomatic treatments, such as corticosteroids to control the effects of the raised pressure within the skull, and medication to help control seizures, as required. Several medicinal products for the treatment of the condition were authorised at the time of submission of the application for orphan designation. Satisfactory argumentation has been submitted by the sponsor to justify the assumption that doxorubicin hydrochloride (drug eluting beads) might be of potential significant benefit for the treatment of glioma, particularly in terms of its new form and route of administration. 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?

Doxorubicin hydrochloride (drug eluting beads) is going to be administered directly into the brain, by injection into the resection margins of the tumour. Therefore, the chemotherapeutic agent (doxorubicin hydrochloride) could be released from the drug eluting beads directly into any possible tumour residue. As a consequence doxorubicin hydrochloride (drug eluting beads) may increase the dose of doxorubicin hydrochloride to the tumour residue, whilst decreasing the side effects due to lower levels of doxorubicin hydrochloride in the blood.

What is the stage of development of this medicine?

The effects of doxorubicin hydrochloride (drug eluting beads) were 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.

Doxorubicin hydrochloride (drug eluting beads) was not authorised anywhere in the world for the treatment of glioma or designated as an orphan medicinal product elsewhere for this condition, at the time of submission.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 10 October 2007 recommending the granting of this 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;
  • 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

Product details for <p>Doxorubicin hydrochloride (drug eluting beads)</p>
Active substanceDoxorubicin hydrochloride (drug eluting beads)
Medicine Name
Disease/conditionTreatment of glioma
Date of decision28/11/2007
OutcomePositive
Orphan decision numberEU/3/07/507

Review of designation

Sponsor’s contact details:

Biocompatibles UK Limited 
Chapman House
Weydon Lane
Farnham
Surrey GU9 8QL
United Kingdom
Tel. +44 1252 732 732
Fax: +44 1252 732 777
E-mail: info@biocompatibles.com

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.