EU/3/19/2153: Orphan designation for the treatment of spinal cord injury

The spinal cord can be injured through accidents or by internal causes such as tumours or bleeding within the spine putting pressure on the spinal cord. The resulting damage to the nerves that run through the cord and that branch out from it can stop the flow of nerve impulses between the brain and the body. This leads to loss of feeling, paralysis and even death, depending on the severity of the injury and where it is located. Patients may also experience loss of control of bladder or bowels, breathing difficulties, blood clots in veins and lungs, and recurring infections.

Spinal cord injury is debilitating and life threatening because it can cause paralysis of the arms and legs and reduces life expectancy.

At the time of designation, spinal cord injury affected approximately 4.46 in 10,000 people in the European Union (EU). This was equivalent to a total of around 231,000 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).

*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 518,400,000 (Eurostat 2019).

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 soon after it is damaged. Patients with spinal cord injury could also have decompression surgery to reduce the pressure on the spine. Other medicines or procedures were used to manage various symptoms such as spasticity (painful muscle spasm) and incontinence.

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with spinal cord injury. Laboratory studies suggest that the medicine may improve the patient’s ability to move more than the authorised treatment. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

This medicine is made of stem cells taken from the bone marrow of the patient. Stem cells can develop into different types of cells. The exact way these stem cells work is not clear but when injected back into the patient’s spine, they are expected to help repair the damaged nerve cells, improving the symptoms of patients with spinal cord injury.

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, the medicine was not authorised anywhere in the EU for the treatment of spinal cord injury 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 21 March 2019, 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.