EU/3/14/1273 - orphan designation for treatment of glioma

autologous dendritic cells pulsed with RNA from glioma stem cells

Overview

On 4 June 2014, orphan designation (EU/03/14/1273) was granted by the European Commission to Epitarget AS, Norway, for autologous dendritic cells pulsed with RNA from glioma stem cells for the treatment of glioma.

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 2.2 in 10,000 people in the European Union (EU). This was equivalent to a total of around 112,000 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 28), Norway, Iceland and Liechtenstein. This represents a population of 511,100,000 (Eurostat 2014).

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 this medicine might be of significant benefit for patients with glioma because early clinical studies showed that the medicine increased progression-free survival (how long patients lived without their disease getting worse) of patients when given in combination with currently authorised treatments. 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?

This medicine is expected to work by activating the patient's immune system (the body's natural defences) so that it attacks and kills the cancer cells.

The medicine is prepared from the patient's own (autologous) immune cells called dendritic cells. These cells are mixed in the laboratory with genetic material from stem cells from the patient's glioma (cells from which the glioma is believed to originate and propagate). When the dendritic cells are injected back into the patient, it is expected that they will help the immune system recognise the patient's glioma cells as 'foreign' and stimulate an immune response against them, helping to slow down or stop the progression of the disease.

What is the stage of development of this medicine?

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

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 9 April 2014 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/03/14/1273: Public summary of opinion on orphan designation of autologous dendritic cells pulsed with RNA from glioma stem cells for the treatment of glioma

Adopted Reference Number: EMA/COMP/228647/2014

Key facts

• Active substance:
  autologous dendritic cells pulsed with RNA from glioma stem cells
• Intended use:
  Treatment of glioma
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/14/1273
• Date of designation:
  04/06/2014
• Sponsor:

  Epitarget AS; Oslo Science Park; Gaustadalléen 21; 0349 Oslo; Norway; E-mail: [e-mail]