EU/3/19/2135: Orphan designation for the treatment of DiGeorge syndrome

Allogeneic cultured postnatal thymus-derived tissue

Table of contents


On 26 February 2019, orphan designation (EU/3/19/2135) 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 DiGeorge syndrome.

Key facts

Active substance
Allogeneic cultured postnatal thymus-derived tissue
Intended use
Treatment of DiGeorge syndrome
Orphan designation status
EU designation number
Date of designation

Enzyvant Therapeutics Ireland Limited
88 Harcourt Street
Dublin 2 D02 DK18
Tel. +353 87 7206017

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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