EU/3/12/1050 - orphan designation for treatment of traumatic spinal cord injury

[2-Cyano-3-cyclopropyl-3-hydroxy-N-(3-methyl-4-trifluoromethylphenyl)prop-2-enamide]

Overview

On 10 October 2012, orphan designation (EU/3/12/1050) was granted by the European Commission to Algiax Pharmaceuticals GmbH, Germany, for [2-cyano-3-cyclopropyl-3-hydroxy-N-(3-methyl-4-trifluoromethylphenyl)prop-2-enamide] for the treatment of traumatic spinal-cord injury.

What is traumatic spinal-cord injury?

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 the death of many of those nerve cells. In the recovery phase, the surviving nerve cells 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 condition that is debilitating in the long-term, because it can cause paralysis of the arms and legs and other health complications involving the functions of sensation and movement. It reduces life expectancy, with around 50% of patients dying in the first month after injury.

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

At the time of designation, traumatic spinal-cord injury affected approximately 3 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 152,000 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 27), Norway, Iceland and Liechtenstein. This represents a population of 506,300,000 (Eurostat 2011).

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 can also have decompression surgery to reduce the pressure on the spine.

The sponsor has provided sufficient information to show that [2-cyano-3-cyclopropyl-3-hydroxy-N-(3-methyl-4-trifluoromethylphenyl)prop-2-enamide] might be of significant benefit for patients with traumatic spinal cord injury, because early studies show that it might improve the outcome of patients by reducing problems with sensation and movement following the injury. 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?

This medicine is expected to have an anti-inflammatory effect by blocking the action of an enzyme called dihydro-orotate dehydrogenase, which is involved in the process of inflammation caused by the immune system's reaction to a traumatic spinal cord injury. Although the inflammation can benefit the central nervous system, it can worsen the damage to body tissue and prevent the recovery of normal functions of sensation and movement. By reducing the inflammation, this medicine is expected to reduce problems with sensation and movement following traumatic spinal-cord injury.

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 the medicinal product in experimental models was ongoing.

At the time of submission, no clinical studies with the medicine in patients with traumatic spinal cord injury had been started.

At the time of submission, the medicinal product 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 5 September 2012 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/3/12/1050: Public summary of opinion on orphan designation: [2-Cyano-3-cyclopropyl-3-hydroxy-N-(3-methyl-4-trifluoromethylphenyl)prop-2-enamide] for the treatment of traumatic spinal-cord injury

Reference Number: EMA/COMP/629399/2012

Key facts

• Active substance:
  [2-Cyano-3-cyclopropyl-3-hydroxy-N-(3-methyl-4-trifluoromethylphenyl)prop-2-enamide]
• Intended use:
  Treatment of traumatic spinal cord injury
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/12/1050
• Date of designation:
  10/10/2012
• Sponsor:

  Algiax Pharmaceuticals GmbH; Max-Planck-Strasse 15a; 40699 Erkrath; Germany; Telephone: [phone]; Telefax: [phone]; E-mail: [e-mail]

Review of designation

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