EU/3/08/553

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Orphan designation

On 3 June 2008, orphan designation (EU/3/08/553) was granted by the European Commission to Gregory Fryer Associates Ltd, United Kingdom, for recombinant fusion protein of circularly-permuted IL-4 and pseudomonas exotoxin A, [IL-4(38-37)-PE38KDEL] for the treatment of glioma.

What is glioma?

Glioma is a type of brain tumour that begins in ‘glial’ cells (the cells that surround and support nerve cells). Patients with gliomas can suffer from different symptoms, depending on the part of the brain where the tumour develops. Glioma is a life-threatening disease.

What are the methods of treatment available?

Treatment for glioma may combine different methods such as surgery, radiotherapy (treatment with radiation) and chemotherapy (medicines used to treat cancer). Other treatments are used to relieve the disease’s symptoms, including corticosteroids that help to reduce pressure within the skull, and medicines that help with the control of seizures (fits). At the time of orphan designation, several medicines were authorised in the European Union for the treatment of glioma and its symptoms.

Recombinant fusion protein of circularly-permuted IL-4 and pseudomonas exotoxin A, [IL-4(38-37)-PE38KDEL] could be of benefit in the treatment of glioma because it seems to have less severe side effects and because it accumulates in the area in the brain where the tumour is developing. These assumptions will need to be confirmed at the time of marketing authorisation, to maintain the orphan status of the medicine.

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

Based on the information provided by the sponsor and previous knowledge of the Committee, glioma was considered to affect approximately 1 in 10,000 persons in the European Union, which, at the time of designation, corresponded to about 50,000 persons.

*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 498,000,000 (Eurostat 2006). This estimate is based on available information and calculations presented by the sponsor at the time of the application.

How is this medicinal product expected to act?

Recombinant fusion protein of circularly-permuted IL-4 and pseudomonas exotoxin A, [IL-4(38-37)-PE38KDEL] is made of a bacterial toxin (exotoxin A) attached to interleukin-4 (IL-4). Exotoxin A is a toxic substance produced by the bacterium Pseudomonas aeruginosa, and IL-4 is a human protein that binds to a specific protein receptor found at higher levels on the surface of tumour cells compared with normal cells. Because it is attached to IL-4, the exotoxin A is concentrated at the site of the tumour, where it kills the tumour cells, reducing the size of the tumour.

What is the stage of development of this medicinal product?

The effects of recombinant fusion protein of circularly-permuted IL-4 and pseudomonas exotoxin A, [IL-4(38-37)-PE38KDEL] have been evaluated in experimental models. At the time of submission of the application for orphan designation, clinical trials in patients with glioma had been completed.

At the time of submission, recombinant fusion protein of circulary-permuted IL-4 and pseudomonas exotoxin A, [IL-4(38-37)-PE38KDEL] was not authorised anywhere in the world for glioma. Orphan designation of recombinant fusion protein of circularly-permuted IL-4 and pseudomonas exotoxin A, [IL-4(38-37)-PE38KDEL] had been granted in the United States for the treatment of glioma.

According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted a positive opinion on 8 April 2008 recommending the granting of the above-mentioned orphan 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 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 a product’s quality, safety and efficacy is necessary before it can be granted a marketing authorisation.

Key facts

Product details for <p>Recombinant fusion protein of circulary-permuted IL-4 and pseudomonas exotoxin A, [IL-4(38-37)-PE38KDEL]</p>
Active substanceRecombinant fusion protein of circulary-permuted IL-4 and pseudomonas exotoxin A, [IL-4(38-37)-PE38KDEL]
Medicine Name
Disease/conditionTreatment of glioma
Date of decision03/06/2008
OutcomePositive
Orphan decision numberEU/3/08/553

Review of designation

Sponsor’s contact details:

Gregory Fryer Associates Ltd
30 St Thomas Place
Cambridgeshire Business Pak
Ely, Cambs
CB7 4EX
United Kingdom
Telephone: +44 1353 645590
Telefax: +44 1353 645599
E-mail: info@gf-associates.co.uk

Patients’ associations contact points:

Cancer BACUP
3 Bath Place
Rivington Street
London
EC2A 3JR
United Kingdom
Telephone: +44 20 76 96 90 03 / 0808 800 1234 (freephone for UK)
Telefax: +44 20 76 96 90 02

Deutsche Krebshilfe e.V.
Buschstrasse 32
53113 Bonn
Postfach 1467
53004 Bonn
Germany
Telephone: +49 2 28 72 99 00
Telefax: +49 22 87 29 90 11
E-mail: deutsche@krebshilfe.de

Ligue Nationale Contre le Cancer
13 Av. de la Grande Armee
75116 Paris
France
Telephone: +33 1 45 00 00 17
Tefefax: +33 1 45 00 63 06
E-mail: ligue@ligue-cancer.net