On 10 March 2005, orphan designation (EU/3/05/264) was granted by the European Commission to TROPHOS SA, France for cholest-4-en-3-one, oxime for the treatment of 5 q spinal muscular atrophy.
The sponsorship was transferred to Roche Registration Limited, United Kingdom, in November 2015.
The sponsorship was transferred to Roche Registration GmbH, Germany in October 2018.
Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in April 2019 on request of the Sponsor.
|Disease / condition||
Treatment of 5q spinal muscular atrophy
|Date of first decision||
|EU designation number||
Sponsor's contact details
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
- European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
- Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;