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.

Key facts

Active substance
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

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

Sponsor's contact details

Audentes Therapeutics Netherlands B.V.
Schiphol Boulevard 359
Noord-Holland 1118 BJ
The Netherlands

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;

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