EU/3/13/1206

On 13 November 2013, orphan designation (EU/3/13/1206) was granted by the European Commission to Neuronax SAS, France, for synthetic 12 amino acid peptide designed after subcommissural organ-spondin for the treatment of spinal cord injury.

What is spinal cord injury?

The spinal cord can be injured through accidents, such as damage to the back, or by internal causes such as tumours or bleeding within the spine putting pressure on the spinal cord. Injury to the spinal cord can damage the nerves that run through the cord and that branch out from it. This can stop the flow of nerve impulses between the brain and the body, resulting in loss of feeling, paralysis and even death, depending upon the severity of the injury and where it is located.

Spinal cord injury is a life-threatening disease that is debilitating in the long-term, because it can cause paralysis of the arms and legs and reduces life expectancy.

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

At the time of designation, spinal cord injury affected less than 4.2 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 215,000 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 512,200,000 (Eurostat 2013).

What treatments are available?

At the time of designation, methylprednisolone (a steroid) was authorised for the treatment of spinal cord injury in some countries in the EU. Methylprednisolone reduces the inflammation and pressure on the spinal cord that can occur after it is damaged. Patients with spinal cord injury could also have decompression surgery to reduce the pressure on the spine.

The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with spinal cord injury because it works in a different way to existing treatments and experimental studies show that it may encourage the survival and regrowth of nerve cells. 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?

Subcommissural organ-spondin (SCO-spondin) is a substance secreted by the brain during its development before birth and is thought to play a role in the development of the nervous system. This medicine is a synthetic version of part of the SCO-spondin molecule. When introduced into the damaged spinal cord it is expected to encourage the survival and re-growth of nerve cells and their connections, thereby reducing the area of damage to the spinal cord and improving its recovery.

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 spinal cord injury had been started.

At the time of submission, synthetic 12 amino acid peptide designed after subcommissural organ-spondin was not authorised anywhere in the EU for spinal cord injury 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 October 2013 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.