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.

Key facts

Active substance
5,7-dichloro-2-((ethylamino)methyl)-8-hydroxy-3-methylquinazolin-4(3H)-one mesilate
Disease / condition
Treatment of multiple system atrophy
Date of first decision
EU designation number

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor's contact details

Pharma Gateway AB
Johanneslundsvagen 2
Hammarby 194 61
Upplands Vaesby Stockholms Lan

Patients' organisations

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.

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