EU/3/11/937

About

On 9 December 2011, orphan designation (EU/3/11/937) was granted by the European Commission to Edison Europe Holding BV, the Netherlands, for alpha-tocotrienol quinone for the treatment of Leigh syndrome.

The sponsorship was transferred to PTC Therapeutics International Limited, Ireland, in November 2019.

Key facts

Active substance
Alpha-tocotrienol quinone
Disease / condition
Treatment of Leigh syndrome
Date of first decision
09/12/2011
Outcome
Positive
EU designation number
EU/3/11/937

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

PTC Therapeutics International Limited
5th floor, 3 Grand Canal Plaza
Grand Canal Street Upper
Dublin 4
D04 EE70
Ireland
E-mail: jtaccoen@ptcbio.com 

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;

How useful was this page?

Add your rating