SCI treatment

What is spinal cord injury?

The spinal cord is a cylindrical extension of the brain that passes through the vertebral canal which 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 intensity 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 are the methods of treatment available?

No satisfactory methods for the treatment of spinal cord injury existed at the time of application.
What is the estimated number of patients affected by the condition*?

According to the information provided by the sponsor, spinal cord injury was considered to affect about 27,600 persons in the European Union.

How is this medicinal product expected to act?

When the spinal cord is injured, there is formation of a scar as normal process of tissue repair. However, the scar will not allow nerves to grow and make new connections that would restore the normal flux of information in the spinal cord. Deferoxamine binds to iron and makes it less available to the tissues. Iron is one of the elements required to produce collagen, the major component of the scars. 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 give an opportunity to the damaged nerves to restore the connections and then the functions.

* Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed based on data from the European Union (EU 25), Norway, Iceland and Lichtenstein. This represents a population of 459,700,000 (Eurostat 2004). This estimate is based on available information and calculations presented by the sponsor at the time of the application.
©EMEA 2007

What is the stage of development of this medicinal product?
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.
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Opinions on orphan medicinal products designations are based on the following cumulative criteria: (i) the seriousness of the condition, (ii) the existence or not of alternative methods of diagnosis, prevention or treatment and (iii) either the rarity of the condition (considered to affect not more than five in ten thousand persons in the Community) or the insufficient return of development investments.

Designated orphan medicinal products are still investigational products which were considered for designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of the quality, safety and efficacy will be necessary before this product can be granted a marketing authorisation.

For more information:
Sponsor’s contact details:
Neuraxo Biopharmaceuticals GmbH
Max-Planck-Straße 15a
D-40699 Erkrath
Germany
Telephone: +49 211 617851 0
Telefax: : +49 211 617851 50
E-mail: info@neuraxo.com
@EMEA 2007

Source - European Medicines Agency

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