EU/3/02/101

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Orphan designation

On 30 April 2002, orphan designation (EU/3/02/101) was granted by the European Commission to PPD Global Ltd, United Kingdom, for pseudomonas exotoxin (domains II/III)-interleukin 13 chimeric protein [hIL13-PE-38QQR (plasmid phuIL13-Tx)] for the treatment of glioma.

The sponsorship was transferred to EUDRAC Limited, United Kingdom, in February 2012.

For a list of the administrative updates to this public summary of opinion, please refer to the PDF document below.

What is glioma?

Tumours that begin in brain tissue are known as primary brain tumours. Primary brain tumours are classified by the type of tissue from which they originate. The most common brain tumours are gliomas, which begin in the glial (supportive) tissue.

Gliomas develop in the brain and patients with gliomas can suffer neurological complications. Gliomas can be debilitating and life-threatening, despite available treatments.

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

At the time of designation, glioma affected approximately 0.8 in 10,000 people in the European Union (EU). This was equivalent to a total of around 30,000 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. At the time of designation, this represented a population of 380,600,000 (Eurostat 2002).

What are the methods of treatment available?

Treatment for glioma depends on a number of factors and may include surgery and radiation therapy. Chemotherapy is also used in some cases. Corticosteroids are used to control symptoms such as a raised pressure in the skull. Other drugs help to control epilepsy, if present. At the time of designation some drugs had already been approved in the European Union for the treatment of some types of gliomas. PE-IL13 may have a new mechanism of action against gliomas. For this reason, it might be of potential significant benefit for the treatment of gliomas.

How is this medicinal product expected to act?

hIL13-PE-38QQR (plasmid phuIL13-Tx) is the combination of a bacterial toxin with a human protein called interleukin 13. The toxin is derived from a bacterium (Pseudomonas aeruginosa). Interleukin 13 interacts with a specific protein receptor found on the surface of cells. Glioma cells have a high number of such protein receptors on their surface compared to normal cells. Thus it is expected that through this mechanism the toxin will target and kill tumour cells.

What is the stage of development of this medicinal product?

The effects of hIL13-PE-38QQR (plasmid phuIL13-Tx) have been studied in experimental models. Clinical trials in patients with glioma had been initiated and were ongoing at the time of designation.

The product had not been marketed in any country at the time of designation. In the United States orphan drug status was granted for the treatment of malignant glioma.

According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 26 March 2002 a positive opinion recommending the grant of the above-mentioned designation.

Opinions on orphan medicinal products designations are based on the following cumulative criteria:
  • the seriousness of the condition,
  • the existence or not of alternative methods of diagnosis, prevention or treatment and 
  • either the rarity of the condition (considered to affect not more than five in ten thousand persons in the Community) or the insufficient return of development investments.

Designated orphan medicinal products are still investigational products which have been considered for 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 will be necessary before this product can be granted a marketing authorisation.

Key facts

Product details for <p>Pseudomonas exotoxin (domains II/III)-interleukin 13 chimeric protein</p>
Active substancePseudomonas exotoxin (domains II/III)-interleukin 13 chimeric protein
Medicine Name
Disease/conditionTreatment of glioma
Date of decision30/04/2002
OutcomePositive
Orphan decision numberEU/3/02/101

Review of designation

Sponsor’s contact details

EUDRAC Limited
Hampden House
Monument Business Park
Chalgrove OX44 7RW
United Kingdom
Tel. +44 (0)1865 893274
Fax +44 (0)1865 893333
E-mail: carole.pugh@eudrac.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.