On 21 August 2020, orphan designation EU/3/20/2311 was granted by the European Commission to Scendea (NL) B.V., Netherlands, for sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate (also known as REN001) for the treatment of mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes (MELAS).

Key facts

Active substance
Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate
Disease / condition
Treatment of mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes
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

Scendea (NL) B.V.
De Cuserstraat 93
1081 CN Amsterdam
The Netherlands
Tel. +31 2089 49169

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