EU/3/07/431 - orphan designation for treatment of glioma

autologous dendritic cells pulsed with autologous tumour cell lysate

Overview

On 15 February 2007, orphan designation (EU/3/07/431) was granted by the European Commission to Dorian Regulatory Affairs BV, the Netherlands, for autologous dendritic cells pulsed with autologous tumour cell lysate for the treatment of glioma.

The sponsorship was transferred to Northwest Biotherapeutics GmbH, Germany, in May 2012.

What is glioma?

Tumours that arise in the 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 arise in the glial (supportive) tissue. Patients affected by gliomas can suffer from severe symptoms of the nervous system, depending on where in the brain the tumour develops. Gliomas are life-threatening.

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).

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*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 500,300,000 (Eurostat 2007).

What treatments are available?

Treatment for glioma depends on a number of factors and encompasses several methods such as 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 some 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. Several products for the treatment of glioma were authorised in the Community at the time of submission of the application for orphan designation.

Autologous dendritic cells pulsed with autologous tumour cell lysate might be of potential significant benefit for the treatment of glioma because they might improve the long-term outcome of the patients. 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?

Autologous dendritic cells pulsed with autologous tumour cell lysate consist of cancer cells of the patient. Once extracted from the patient, the cancer cells are modified to increase their ability to create an immune response. The cells are then killed and given to the patient with the aim of activating the patient's immune system against any cancer cells, which could remain in the body after surgery, thus reducing the possibility of a relapse.

What is the stage of development of this medicine?

The effects of autologous dendritic cells pulsed with autologous tumour cell lysate 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.

Autologous dendritic cells pulsed with autologous tumour cell lysate were not authorised anywhere worldwide for treatment of glioma or designated as an 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 10 January 2007 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.

EU/3/07/431: Public summary of positive opinion for orphan designation of autologous dendritic cells pulsed with autologous tumour cell lysate for the treatment of glioma

Adopted Reference Number: EMA/COMP/96767/2008 Rev. 1

Key facts

• Active substance:
  autologous dendritic cells pulsed with autologous tumour cell lysate
• Intended use:
  Treatment of glioma
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/07/431
• Date of designation:
  15/02/2007
• Sponsor:

  Northwest Biotherapeutics GmbH; Deutscher Platz 5; 04103 Leipzig; Germany; Tel. [phone]; Fax [phone]