EU/3/19/2136: Orphan designation for the treatment of CHARGE syndrome

Allogeneic cultured postnatal thymus-derived tissue

Table of contents

Overview

On 26 February 2019, orphan designation (EU/3/19/2136) was granted by the European Commission to Enzyvant Therapeutics Ireland Limited, Ireland, for allogeneic cultured postnatal thymus-derived tissue (also known as RVT-802) for the treatment of CHARGE syndrome.

Key facts

Active substance
Allogeneic cultured postnatal thymus-derived tissue
Intended use
Treatment of CHARGE syndrome
Orphan designation status
Positive
EU designation number
EU/3/19/2136
Date of designation
26/02/2019
Sponsor

Enzyvant Therapeutics Ireland Limited
88 Harcourt Street
Dublin 2 D02 DK18
Ireland
Tel. +353 87 7206017
E-mail: info@enzyvant.com

Documents related to this orphan designation evaluation

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

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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