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 (Inetagugene geperpavec)
Table of contents
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 (Inetagugene geperpavec)
|
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 |
Krystal Biotech Netherlands B.V.
|
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 |
---|---|
January 2023 | The sponsorship was transferred from IDEA Innovative Drug European Associates (Ireland) Limited to Krystal Biotech Netherlands B.V. |
July 2022 | The sponsor’s address was updated in July 2022. |
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: