EU/3/12/1029

  • Email
  • Help

Orphan designation

Please note that this product was withdrawn from the Community register of designated orphan medicinal products in July 2013 on request of the sponsor.

On 9 August 2012, orphan designation (EU/3/12/1029) was granted by the European Commission to Abbott Laboratories, United Kingdom, for humanised monoclonal antibody against epidermal-growth-factor receptor for the treatment of glioma.

The sponsorship was transferred to AbbVie Ltd, United Kingdom, in January 2013.

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

What is glioma?

Glioma is a type of brain tumour that affects the ‘glial’ cells (the cells that surround and support the nerve cells). Patients with glioma can have severe symptoms, but the types of symptoms experienced depend on where the tumour develops in the brain. Symptoms can include headaches, nausea (feeling sick), loss of appetite, vomiting, and changes in personality, mood, mental capacity and concentration. About a fifth of patients with glioma have seizures (fits) for months or years before the disease is diagnosed.

Glioma is a long-term debilitating and life-threatening disease because of the severe damage to the brain and is associated with poor long-term survival.

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 51,000 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 509,000,000 (Eurostat 2012).

What treatments are available?

At the time of designation, several medicines were authorised for the treatment of glioma in the EU. Treatments for glioma included surgery, radiotherapy (treatment with radiation), and chemotherapy (medicines to treat cancer) to improve survival. Patients also received treatments for the symptoms of glioma, including corticosteroids to reduce pressure within the skull and medicines to prevent seizures.

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients on the basis of studies in experimental models showing that the medicine in combination with temozolomide (an anticancer medicine authorised in the EU for the treatment of glioma) and radiotherapy might block tumour growth better than temozolomide and radiotherapy alone. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

The medicine is a monoclonal antibody, a type of protein designed to recognise and target a specific structure found in certain cells. The medicine targets epidermal-growth-factor receptors (EGFR) found on the surface of some tumour cells. These receptors play an important role in the growth, progression and spread of the cancer. By attaching to and blocking their action, the medicine is expected to help slow down the growth of the cancer.

What is the stage of development of this medicine?

The effects of the medicine have been evaluated in experimental models.

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

At the time of submission, the medicine was not authorised anywhere in the EU for glioma or designated as an orphan medicinal product elsewhere for this condition. Orphan designation of the medicine had been granted in the United States for glioblastoma multiforme, a type of glioma.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 11 July 2012 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>Humanised monoclonal antibody against epidermal-growth-factor receptor</p>
Active substanceHumanised monoclonal antibody against epidermal-growth-factor receptor
Medicine Name
Disease/conditionTreatment of glioma
Date of decision09/08/2012
OutcomeWithdrawn
Orphan decision numberEU/3/12/1029

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor’s contact details

AbbVie Ltd
Abbott House
Vanwall Business Park
Vanwall Road
Maidenhead
Berkshire
SL6 4XE
United Kingdom
Tel. +44 (0)1628 644501
Fax +44 (0)1628 672624
E-mail: orphandrugs.enquiries@abbvie.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.