EU/3/14/1248

On 19 February 2014, orphan designation (EU/3/14/1248) was granted by the European Commission to Bionure Farma SL, Spain, for N-({Carbamoylmethyl-[3-(2-oxo-pyrrolidin-1-yl)-propyl]-carbamoyl}-methyl)-2-[2-(2-fluoro-phenyl)-ethylamino]-N-isobutyl-acetamide for the treatment of optic neuritis.

For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.

What is optic neuritis?

Optic neuritis is a disease characterised by inflammation of the optic nerve, the nerve at the back of the eye that transmits signals to the brain. Optic neuritis is caused by an overactive immune system (the body’s natural defences) which attacks the optic nerve leading to inflammation and death of the nerve cells. Symptoms often affect only one eye and include loss of vision, loss of colour vision and pain around the eye. Optic neuritis often develops in people with diseases of the immune system.

Optic neuritis is a long-term debilitating disease because of the visual loss and development of blindness.

What is the estimated number of patients?

At the time of designation, optic neuritis affected approximately 0.5 in 10,000 people in the European Union (EU).This was equivalent to a total of around 26,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased 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. At the time of designation, this represented a population of 512,900,000 (Eurostat 2014).

What treatments are available?

At the time of designation, there were no satisfactory methods specifically authorised in the EU for the treatment of optic neuritis. Only medicines to relieve the inflammation and symptoms were available, including corticosteroids (anti-inflammatory medicines) and medicines that reduce the activity of the immune system (the body’s natural defences).

How is this medicine expected to work?

The way this medicine works is not clear, but it is expected to cross the blood-brain barrier, which separates the blood from the brain tissue. In the brain, this medicine is thought to protect nerve cells from damage and it has been shown to attach to certain proteins called SGK2, which help nerve cells to survive.

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 optic neuritis had been started.

At the time of submission, this medicine was not authorised anywhere in the EU for optic neuritis 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 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.

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