EU/3/17/1971

Table of contents

About

On 17 January 2018, orphan designation (EU/3/17/1971) was granted by the European Commission to Edison Orphan Pharma BV, The Netherlands, for vatiquinone (also known as alpha-tocotrienol quinone) for the treatment of RARS2 syndrome.

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

Key facts

Active substance
Vatiquinone
Disease / condition
Treatment of RARS2 syndrome
Date of first decision
17/01/2018
Outcome
Positive
EU designation number
EU/3/17/1971

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;

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