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.
- 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 sensibility, 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 is equivalent to a total of around 32,000 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 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 25), Norway, Iceland and Liechtenstein. This represents a population of 459,700,000 (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 to 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 were 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.
According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 9 September 2004 a positive opinion recommending the grant of the above-mentioned 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.
|Name||Language||First published||Last updated|
|EU/3/04/231: Public summary of positive opinion for orphan designation of deferoxamine mesilate for the treatment of traumatic spinal cord injury||(English only)||21/09/2009|
|Active substance||Deferoxamine mesilate|
|Disease/condition||Treatment of traumatic spinal cord injury|
|Date of decision||20/10/2004|
|Orphan decision number||EU/3/04/231|
Review of designation
Sponsor’s contact details:
SCT Spinal Cord Therapeutics GmbH
Telephone: +49 211 617851 0
Telefax: : +49 211 617851 50
Patients’ associations contact points:
Associazione Luca Coscioni
via di Torre Argentina
76 - 00186 Roma
Telephone: +39 06 68 97 91
Telefax: +39 06 68 80 53 96
Danish Spinal Cord Injuries Association
Hans Knudsens Plads 1 A
DK 2100 København Ø
Telephone: +45 39 29 35 55
Telefax: +45 39 29 39 48
I.W.A. – Irish Wheelchair Association
Telephone: +353 01 8186 400
Telefax: +33 01 8333 873