EU/3/10/754:

Traumatic spinal cord injury is damage to the spinal cord caused by an accident, such as a blow to the back. Injury to the spinal cord can damage the nerves that run through the cord and branch out from it. This can stop the flow of nerve impulses between the brain and the rest of the body, resulting in the loss of sensation, paralysis and even death, depending on the severity and location of the injury.

The development of traumatic spinal cord injury can be divided into two phases: the acute phase and the recovery phase. During the acute phase (lasting for a few weeks after the injury) a process of inflammation starts, in which the damage spreads to the nerve cells surrounding the original site of injury, leading to many of the nerve cells around the site of the injury dying. In the recovery phase, the surviving nerves recover some of their function. The improvement usually continues for up to one year, after which the patient's condition tends not to improve any further.

Traumatic 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.

At the time of designation, traumatic spinal cord injury affected approximately 4.2 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 213,000 people, and is below the threshold 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 27), Norway, Iceland and Liechtenstein. This represents a population of 506,500,000 (Eurostat 2010).

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 happen after it is damaged. Patients with spinal cord injury can also have surgery to reduce the pressure on the spine.

The sponsor has provided sufficient information to show that heparin-activated recombinant human fibroblast growth factor 1 (on a biodegradable device made from alpha-calcium sulphate hemihydrate) might be of significant benefit for patients with traumatic spinal cord injury because it works in a different way to existing treatments, and because early studies in experimental models indicate that it might stimulate the repair and regrowth of the nerves during the recovery phase. These assumptions will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

This medicine is made of a device that contains several channels coated with fibroblast growth factor 1, a natural substance that stimulates the development of cells, including nerve cells. The medicine also contains heparin, which is used to stimulate the activity of the growth factor. The medicine is expected to be implanted during surgery. Before implantation, nerves from another part of the body (generally the calf) will be inserted into the device. The surgeon will then place the device at the site of injury so that the calf nerves form a bridge across the site of injury in the spinal cord. The device is expected to stimulate the nerves at either side of the injury to start to grow and form new connections across the site of injury. This is expected to start to repair the injury, eventually leading to some recovery of sensation and movement. The device is expected to dissolve away slowly after being implanted in the body.

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 traumatic spinal cord injury had been started.

At the time of submission, this medicine was not authorised anywhere in the EU for traumatic 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 8 April 2010 recommending the granting of this designation.

• 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.