EU/3/19/2228: Orphan designation for the treatment of multiple system atrophy
Table of contents
On 16 December 2019, orphan designation EU/3/19/2228 was granted by the European Commission to Alterity Therapeutics UK Limited, United Kingdom, for 5,7-dichloro-2-((ethylamino)methyl)-8-hydroxy-3-methylquinazolin-4(3H)-one mesilate (also known as PBT434) for the treatment of multiple system atrophy.
The sponsorship was transferred to Pharma Gateway AB, Sweden, in October 2020.
Treatment of multiple system atrophy
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Pharma Gateway AB
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
Documents related to this orphan designation evaluation
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.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: