EU/3/19/2225: Orphan designation for the treatment of autosomal recessive congenital ichthyosis

Replication-incompetent, non-integrating, herpes simplex virus 1 vector expressing the human transglutaminase-1 enzyme

Overview

On 13 November 2019, orphan designation EU/3/19/2225 was granted by the European Commission to IDEA Innovative Drug European Associates (Ireland) Limited, Ireland, for replication-incompetent, non-integrating, herpes simplex virus 1 vector expressing the human transglutaminase-1 enzyme for the treatment of autosomal recessive congenital ichthyosis.

Key facts

Active substance
Replication-incompetent, non-integrating, herpes simplex virus 1 vector expressing the human transglutaminase-1 enzyme
Intended use
Treatment of autosomal recessive congenital ichthyosis
Orphan designation status
Positive
EU designation number
EU/3/19/2225
Date of designation
13/11/2019
Sponsor

IDEA Innovative Drug European Associates (Ireland) Limited

Unit 13 Classon House
Dundrum Business Park
Dundrum Road
Dublin 14
D14 W9Y3
Ireland

E-mail: inquires@krystalbio.com

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product it it is approved for marketing authorisation.

Update history

Date Update
July 2022 The sponsor’s address was updated in July 2022.

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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