EU/3/20/2319: Orphan designation for the treatment of long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency

Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate

Overview

On 21 August 2020, orphan designation EU/3/20/2319 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 long-chain 3-hydroxyacyl-CoA dehydrogenase deficiency.

Key facts

Active substance
Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate
Intended use
Treatment of long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency
Orphan designation status
Positive
EU designation number
EU/3/20/2319
Date of designation
21/08/2020
Sponsor

Scendea (NL) B.V.
De Cuserstraat 93
1081 CN Amsterdam
Noord-Holland
Netherlands
Tel. +31 2089 49169
E-mail: orphan@scendea.com

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

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.

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:

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