EU/3/20/2368 - orphan designation for treatment of glioma

human interleukin 12 fused with immunoglobulin G4 C-terminal Fc fragment

Overview

On 9 December 2020, orphan designation EU/3/20/2368 was granted by the European Commission to VH Regulatory Consulting GmbH & Co. KG, Germany, for human interleukin 12 fused with immunoglobulin G4 C-terminal Fc fragment for the treatment of glioma.

What is Prader-Willi syndrome?

Prader-Willi syndrome is an inherited condition caused by defects in specific regions of chromosome 15. This causes a wide range of symptoms, some of which can appear at birth, such as feeding problems, small size and reduced muscle strength. During childhood further symptoms develop, including increased appetite leading to constant eating and severe obesity, short stature, incomplete sexual development, learning difficulties and behavioural problems, such as aggression and stubbornness.

Prader-Willi syndrome is a life-long debilitating and life-threatening disease because of its serious symptoms, particularly learning difficulties, behavioural problems and obesity.

What is the estimated number of patients affected by the condition?

At the time of designation, glioma affected approximately 2.6 in 10,000 people in the European Union (EU). This was equivalent to a total of around 135,000 people¹, 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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¹For 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, Iceland, Liechtenstein, Norway and the United Kingdom. This represents a population of 519,200,000 (Eurostat 2020).

 

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). 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 results from laboratory studies suggest that adding the medicine to temozolomide (chemotherapy) and radiotherapy leads to greater benefits.

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 contains interleukin-12 (IL-12), a protein that activates the immune system (the body’s natural defences), and is expected to work by helping the immune system to fight the cancer. In this medicine, IL-12 is combined with a type of protein called immunoglobulin G4 fragment, which helps IL-12 stay active for longer. The medicine is expected to be delivered directly into the tumour.

What is the stage of development of this medicine?

At the time of submission of the application for orphan designation, the evaluation of the effects of the medicine in experimental models was ongoing.

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 the treatment of glioma or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 5 November 2020, 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/3/20/2368: Public summary of opinion on orphan designation: Human interleukin 12 fused with immunoglobulin G4 C-terminal Fc fragment for the treatment of glioma

Reference Number: EMADOC-628903358-3723

Key facts

• Active substance:
  human interleukin 12 fused with immunoglobulin G4 C-terminal Fc fragment
• Intended use:
  Treatment of glioma
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/20/2368
• Date of designation:
  09/12/2020
• Sponsor:

  VH Regulatory Consulting GmbH & Co. KG; Herderner Strasse 42; 79801 Hohentengen Am Hochrhein; Baden-Wuerttemberg; Germany; Tel. [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.