EU/3/07/504

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Orphan designation

On 29 November 2007, orphan designation (EU/3/07/504) was granted by the European Commission to CellMed AG, Germany, for irinotecan hydrochloride (drug-eluting beads) for the treatment of glioma.

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

What are gliomas?

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 originate. 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. Gliomas are life-threatening.

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

At the time of designation, gliomas 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 of gliomas 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 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 irinotecan hydrochloride (drug-eluting beads) might be of potential significant benefit for the treatment of glioma, particularly in terms of its new 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?

Irinotecan hydrochloride (drug-eluting beads) is to be given directly into the brain, by injection into the resection margin of the tumour. Therefore the chemotherapeutic agent (irinotecan hydrochloride) would be released from the drug-eluting beads directly into the tumour. As a consequence, irinotecan hydrochloride (drug-eluting beads) may maximise the dose of irinotecan hydrochloride to the tumour whilst decreasing the systemic side effects due to lower blood levels of irinotecan hydrochloride.

What is the stage of development of this medicine?

The effects of irinotecan hydrochloride (drug-eluting beads) 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.

Irinotecan hydrochloride (drug-eluting beads) was not authorised anywhere worldwide 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>Irinotecan hydrochloride (drug-eluting beads)</p>
Active substanceIrinotecan hydrochloride (drug-eluting beads)
Medicine Name
Disease/conditionTreatment of glioma
Date of decision28/11/2007
OutcomePositive
Orphan decision numberEU/3/07/504

Review of designation

Sponsor’s contact details:

Biocompatibles UK
Limited Chapman House
Weydon Lane
Farnham
Surrey
GU9 8QL
United Kingdom
Tel. +44 (0)1252 732732
Fax +44 (0)1252 732777
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.