On 29 July 2014, orphan designation (EU/3/14/1296) was granted by the European Commission to Fondazione Salvatore Maugeri Clinica del Lavoro e della Riabilitazione, Italy, for adeno-associated viral vector serotype 9 containing the human cardiac calsequestrin gene for the treatment of catecholaminergic polymorphic ventricular tachycardia.
The sponsorship was transferred to Voisin Consulting S.A.R.L., France, in April 2015 and to Audentes Therapeutics UK Ltd, United Kingdom, in November 2015.
The sponsorship was transferred to Audentes Therapeutics Netherlands B.V., Netherlands in May 2019.
EU/3/14/1296: Public summary of opinion on orphan designation: Adeno-associated viral vector serotype 9 containing the human cardiac calsequestrin gene for the treatment of catecholaminergic polymorphic ventricular tachy... (PDF/137.9 KB)
First published: 01/09/2014
Last updated: 27/05/2015
Adeno-associated viral vector serotype 9 containing the human cardiac calsequestrin gene
|Disease / condition||
Treatment of catecholaminergic polymorphic ventricular tachycardia
|Date of first decision||
|EU designation number||
Review of designation
Sponsor's contact details
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;