EU/3/16/1619 - orphan designation for treatment of non-infectious uveitis

DNA plasmid encoding a recombinant fusion protein consisting of the extracellular domain of human TNFα p55 receptor linked to the human IgG1 Fc domain

Overview

On 17 February 2016, orphan designation (EU/3/16/1619) was granted by the European Commission to Eyevensys SA, France, for DNA plasmid encoding a recombinant fusion protein consisting of the extracellular domain of human TNFα p55 receptor linked to the human IgG1 Fc domain for the treatment of non-infectious uveitis.

What is non-infectious uveitis?

Uveitis is inflammation of the uvea, the middle layer of the eye, just beneath the white part of the eye. The inflammation can affect one or both eyes, and may cause discomfort, pain, and blurring of vision. Non-infectious uveitis is usually caused by the body's immune system (the body's natural defences) attacking normal tissue and not by an infection.

Non-infectious uveitis is a long-term debilitating disease because it may lead to partial or complete loss of vision (blindness).

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

At the time of designation, non-infectious uveitis affected approximately 4.6 in 10,000 people in the European Union (EU). This was equivalent to a total of around 236,000 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 513,700,000 (Eurostat 2016).

What treatments are available?

At the time of designation, several medicines were authorised in Member States of the EU for the treatment of non-infectious uveitis. The first treatment option was corticosteroids, which were used to reduce the inflammation by lowering the activity of the immune system. Other immunosuppressant agents (that reduce the activity of the immune system) such as ciclosporin were also authorised for use in non-infectious uveitis.

The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with non-infectious uveitis because, since the medicine is expected to be given as an injection into the eye, it is expected to have only a local effect in the eye. Use of this medicine may reduce the need to use corticosteroids that have effects on the whole body. These assumptions 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 contains genetic material responsible for making the TNF? p55 receptor. This receptor is normally present on the surface of cells of the immune system and attaches to TNF?, a substance that plays a key role in regulating inflammatory processes in non-infectious uveitis. The medicine is expected to be delivered into cells in the eye, where it produces the TNF? p55 receptor. These new receptors attach to TNF? thereby preventing it from attaching to the natural receptor on immune cells and stopping its effect. This is expected to reduce symptoms of inflammation.

The medicine will be given by electrotransfer, a technique where the medicine is first injected into the eye, after which electric pulses are applied to the eye. This will disrupt the cell walls in the eye and allow the medicine to enter cells where it becomes active.

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 this medicine in experimental models was ongoing.

At the time of submission, no clinical trials with the medicine in patients with non-infectious uveitis had been started.

At the time of submission, the medicine was not authorised anywhere in the EU for non-infectious uveitis or designated as an orphan medicinal product elsewhere for non-infectious uveitis.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 21 January 2016 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/3/16/1619: Public summary of opinion on orphan designation: DNA plasmid encoding a recombinant fusion protein consisting of the extracellular domain of human TNFα p55 receptor linked to the human IgG1 Fc domain for t...

Adopted Reference Number: EMA/COMP/61236/2016

Key facts

• Active substance:
  DNA plasmid encoding a recombinant fusion protein consisting of the extracellular domain of human TNFα p55 receptor linked to the human IgG1 Fc domain
• Intended use:
  Treatment of non-infectious uveitis
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/16/1619
• Date of designation:
  17/02/2016
• Sponsor:

  PulseSight Therapeutics

Review of designation

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