EU/3/06/418

  • Email
  • Help

Orphan designation

On 30 October 2006, orphan designation (EU/3/06/418) was granted by the European Commission to BCG (Europe) Ltd, United Kingdom, for iodine (131I) anti-tenascin monoclonal antibody 81C6 for the treatment of glioma.

The sponsorship was transferred to The Weinberg Group LLC, United Kingdom, in June 2007, to The Weinberg Group Limited, United Kingdom, in March 2009 and subsequently to Biological Consulting Europe Ltd, United Kingdom, in December 2010.

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 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 equivalent to a total of around 46,000 people, and is below the threshold for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and 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. This represents a population of 459,700,000 (Eurostat 2004).

What treatments are 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.

Satisfactory argumentation has been submitted by the sponsor to justify the assumption that iodine (131I) anti-tenascin monoclonal antibody 81C6 might be of potential significant benefit for the treatment of glioma, mainly because it may provide a major benefit to patient care. 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?

The product is a radioactive element (iodine 131) attached to a protein, which specifically binds to glioma cells. Iodine-131 can give off radiation, which in turn damages and kills cells, especially those that are dividing, such as cancer cells. Thus, it is expected that iodine (131I) anti-tenascin monoclonal antibody 81C6 will build up in the glioma cancer cells, and then kill these cells with the radiation produced by the radioactive iodine contained in the molecule.

What is the stage of development of this medicine?

The effects of iodine (131I) anti-tenascin monoclonal antibody 81C6 were evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials in patients with glioma were ongoing.

Iodine (131I) anti-tenascin monoclonal antibody 81C6 was not authorised anywhere worldwide for the treatment of glioma, at the time of submission. Orphan designation of iodine (131I) anti-tenascin monoclonal antibody 81C6 was granted in the United States for the treatment of primary brain tumours.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 4 October 2006 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 Community) 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>Iodine (131I) anti-tenascin monoclonal antibody 81C6</p>
Active substanceIodine (131I) anti-tenascin monoclonal antibody 81C6
Medicine Name
Disease/conditionTreatment of glioma
Date of decision29/10/2006
OutcomePositive
Orphan decision numberEU/3/06/418

Review of designation

Sponsor’s contact details

Biological Consulting Europe Ltd
Logan Building
Roslin BioCentre
Roslin, Midlothian, EH25 9TT
United Kingdom
Tel. +44 (0)13 1440 6480
Fax +44 (0)13 1440 6486
E-mail: a.cameron@bioconsulteurope.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.