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Orphan designation

On 4 December 2007, orphan designation (EU/3/07/511) was granted by the European Commission to MAPREG SAS, France, for 3-methoxy-pregnenolone for the treatment of spinal cord injury.

What is spinal cord injury?

The spinal cord is a cylindrical extension of the brain, contained within the bones of the spine. The spinal cord gives origin to the nerves that send information (sensations) to the brain, and distribute orders to the various parts of the body (mostly to the muscles). The spinal cord is protected by the bones of the spine, but it can be damaged by any external injury (trauma to the back) or by internal causes (a tumour or bleeding, for example). Injury to the spinal cord can damage the nerves, and stop the connection between the brain and the parts of the body that either receive orders from the brain or send information to it. Stopping this communication, results in lack of sensitivity, paralysis and even death, depending upon the intensity and level of the lesion and the structures damaged.
Spinal cord injury is life-threatening and chronically debilitating, due to increased mortality and severe complications (e.g. paralysis of the legs or of all limbs, respiratory difficulties, blood clots in the veins and the lungs, and repeated infections of the kidneys, the respiratory system and the urinary tract.

What are the methods of treatment available?

No satisfactory methods exist that were authorised at the time of application. Surgical intervention is often performed to decrease the pressure over the spine (decompression), but its role is controversial. High doses of methylprednisolone are also sometimes given.


Based on the information provided by the sponsor and previous knowledge of the Committee, spinal cord injury was considered to affect between 2.2 and 4.2 in 10,000 persons in the European Union, which, at the time of designation, corresponded to about 110,000-209,000 persons.


How is this medicinal product expected to act?

3-methoxy-pregnelonone is derived from a natural hormone (a substance that circulates in the blood and modifies bodily functions at a distance), called pregnenolone. 3-methoxy-pregnenolone is expected to help spinal cord cells, which are damaged after spinal cord injury occurs, to recover their structure and eventually their function.

What is the stage of development of this medicinal product?

At the time of submission of the application for orphan designation, no clinical trials in patients with spinal cord injury were initiated.

The medicinal product was not authorised anywhere in the world for the treatment of 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 10 October 2006 a positive opinion recommending the grant of the above-mentioned designation.

  • the seriousness of the condition, 


Key facts

Product details for <p>3-methoxy-pregnenolone</p>
Active substance3-methoxy-pregnenolone
Medicine Name
Disease/conditionTreatment of spinal cord injury
Date of decision04/12/2007
Orphan decision numberEU/3/07/511

Review of designation


Sponsor’s contact details:

CHU Bicêtre Bat Paul Langevin
78 rue du Général Leclerc
94275 Le Kremlin-Bicêtre Cedex
Telephone: +33 1 49 59 18 77
Telefax: +33 1 49 59 92 03

Patients' organisations:

