EU/3/04/231 - orphan designation for treatment of traumatic spinal cord injury

Deferoxamine mesilate

Overview

Please note that this product was withdrawn from the Community register of designated orphan medicinal products in February 2013 on request of the sponsor.

On 20 October 2004, orphan designation (EU/3/04/231) was granted by the European Commission to Neuraxo Biopharmaceuticals GmbH, Germany, for deferoxamine mesilate for the treatment of traumatic spinal-cord injury.

Neuraxo Biopharmaceuticals GmbH changed name to SCT Spinal Cord Therapeutics GmbH in May 2009.

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

What is spinal-cord injury?

The spinal cord is a cylindrical extension of the brain that passes through the vertebral canal that is formed by the bones of the spine. The spinal cord is formed by nerve fibres that send information (sensations) to the brain and distribute orders from it to the various parts of the body (mostly to the muscles). The spinal cord is protected by the spine bones, but it can be damaged by any injury acting directly on it or indirectly through surrounding structures (bones, vessels, etc.). An injury to the spinal cord could damage the nerves and therefore stop the connection between the brain and the distal structures of the body, resulting in lack of sensation, paralysis and even death, depending on the extent and level of the lesion and the structures damaged.

Spinal-cord injury is life-threatening and chronically debilitating, due to the severe complications (e.g. paralysis of all four limbs, respiratory difficulties, blood clots, infections etc.).

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

At the time of designation, spinal-cord injury affected approximately 0.6 in 10,000 people in the European Union (EU). This was equivalent to a total of around 28,000 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 25), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 464,200,000 (Eurostat 2004).

What treatments are available?

No satisfactory methods for the treatment of spinal-cord injury existed at the time of application.

How is this medicine expected to work?

When the spinal cord is injured, the normal process of tissue repair results in the formation of a scar. However, the scar will not allow nerve growth and the formation of new connections that would restore the normal flux of information in the spinal cord. Deferoxamine binds to iron, one of the elements required to produce collagen, the major component of scar tissue, making it less available to the tissues. Therefore, deferoxamine would stop scar formation by decreasing the availability of iron, which is necessary for the production of the scar. The inhibition of the production of the scar would provide an opportunity for the damaged nerves to restore first the connections and then the functions.

What is the stage of development of this medicine?

The effects of deferoxamine mesilate have been evaluated in experimental models.

Deferoxamine mesilate was not marketed anywhere worldwide for spinal-cord injury or designated as orphan medicinal product elsewhere for this condition, at the time of submission.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 9 September 2004 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 Community) 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/3/04/231: Public summary of positive opinion for orphan designation of deferoxamine mesilate for the treatment of traumatic spinal-cord injury

AdoptedReference Number: EMEA/COMP/178558/2004 Rev. 3

Key facts

• Active substance:
  Deferoxamine mesilate
• Intended use:
  Treatment of traumatic spinal cord injury
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/04/231
• Date of designation:
  20/10/2004
• Sponsor:

  SCT Spinal Cord Therapeutics GmbH; Max-Planck-Straße 15a; D-40699 Erkrath; Germany; Tel. [phone]; Fax [phone]; E-mail: [e-mail]