EU/3/15/1443 - orphan designation for treatment of glioma

recombinant human glutamate oxaloacetate transaminase 1

Overview

Please note that this product was withdrawn from the Union Register of orphan medicinal products in March 2021 on request of the Sponsor.

On 12 February 2015, orphan designation (EU/3/15/1443) was granted by the European Commission to Impasara Ltd, United Kingdom, for recombinant human glutamate oxaloacetate transaminase 1 for the treatment of glioma.

The sponsorship was transferred to Dr. Regenold GmbH, Germany, in October 2015.

In July 2017, Dr. Regenold GmbH Development Regulatory Market Access changed name to Dr. Regenold GmbH.

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 one 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 less than 2.5 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 128,000 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 28), Norway, Iceland and Liechtenstein. This represents a population of 512,900,000 (Eurostat 2015).

What treatments are available?

At the time of designation, several medicines were authorised for the treatment of glioma in the EU. Treatments 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 with glioma because studies in experimental models showed that the medicine, when given with another medicine for glioma, temozolomide, might improve the survival of patients with the condition. 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?

In glioma patients, the glioma cells release high levels of glutamate into some parts of the brain. Glutamate is a substance important for brain function but if too much is present it can cause death of nerve cells and thus promote the growth of glioma cells. The medicine is made up of 'glutamate oxaloacetate transaminase', an enzyme normally found in the blood that transforms glutamate into another compound that is not toxic to the brain. By reducing the levels of glutamate in the blood the levels of glutamate in the brain are also expected to decrease. This is expected to slow down the growth of the glioma.

The glutamate oxaloacetate transaminase in this medicine is made by a method known as 'recombinant DNA technology': it is made by cells into which a gene (DNA) has been introduced that makes them able to produce the protein. The medicine is to be delivered by injection under the skin.

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, no clinical trials with the medicine in patients with glioma had been started.

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 January 2015 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/3/15/1443: Public summary of opinion on orphan designation: Recombinant human glutamate oxaloacetate transaminase 1 for the treatment of glioma

Adopted Reference Number: EMA/COMP/26647/2015

Key facts

• Active substance:
  recombinant human glutamate oxaloacetate transaminase 1
• Intended use:
  Treatment of glioma
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/15/1443
• Date of designation:
  12/02/2015
• Sponsor:

  Dr. Regenold GmbH; Zöllinplatz 4; 79410 Badenweiler; Germany; Tel. [phone]; Fax [phone]; E-mail: [e-mail]

Review of designation

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