EU/3/03/184

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Orphan designation

Please note that this product was withdrawn from the Community register of designated orphan medicinal products in March 2013 on request of the sponsor.

On 14 January 2004, orphan designation (EU/3/03/184) was granted by the European Commission to Merck KGaA, Germany, for cilengitide for the treatment of glioma.

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 being gliomas, which begin in the glial (supportive) tissue.

Gliomas represent a potentially debilitating and life-threatening condition, with symptoms being influenced by which regions of the brain are affected. Patients affected by gliomas can suffer from medical problems of the nervous system, depending on where in the brain the tumour develops.

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 46,000 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 25), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 464,200,000 (Eurostat 2004).

What treatments are available?

Treatment for gliomas depends on a number of factors and may include surgery, radiotherapy (using high-dose X-rays or other high-energy rays to kill cancer cells) or chemotherapy (using drugs to kill cancer cells), as well as symptomatic treatments. Symptomatic treatments include certain steroid hormones (corticosteroids) to control the effects of raised pressure within the skull, and medication to help control seizures, as required. Methods of treatment of glioma were authorised at the time of submission of the application for orphan designation.

Cilengitide might be of potential significant benefit for the treatment of gliomas. The main reason is that cilengitide may offer a new way of killing the cancer cells and stopping tumour growth in these patients. The 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?

Cilengitide can block certain proteins called αvβ3 and αvβ5 integrins. Integrins are found on the surface of cells, including certain gliomas. The αvβ3 and αvβ5 integrins play a key role when new blood vessels are made. Cancer cells require a rich network of blood vessels in their vicinity to grow

Furthermore, integrins may also help the glioma cells to spread into the surrounding tissue of the brain. By blocking the growth of vessels supplying blood to the tumour, cilengitide might stop tumour growth.

What is the stage of development of this medicine?

The evaluation of the effects of cilengitide in experimental models is ongoing. At the time of submission of the application for orphan designation, clinical trials in patients with glioma were ongoing.

Cilengitide was not marketed anywhere worldwide for treatment of glioma or designated as 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 5 December 2003 recommending the granting of this designation.

Opinions on orphan medicinal products designations are based on the following cumulative 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.

Name Language First published Last updated
EU/3/03/184: Public summary of positive opinion for orphan designation of cilengitide for the treatment of glioma (English only) 08/07/2004 09/04/2013

Key facts

Product details for <p>Cilengitide</p>
Active substanceCilengitide
Medicine Name
Disease/conditionTreatment of glioma
Date of decision14/01/2004
OutcomeWithdrawn
Orphan decision numberEU/3/03/184

Review of designation

Sponsor’s contact details

Merck KGaA
Frankfurter Strasse 250
D-64293 Darmstadt
Germany
Tel. +49 6151 72 0
Fax +49 6151 72 2000
E-mail: service@merck.de

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.