EU/3/14/1282: Orphan designation for the treatment of Usher syndrome
Mixture of two adeno-associated viral vectors serotype 8 containing the 5'-half sequence of human MYO7A gene and the 3'-half sequence of human MYO7A gene
Table of contents
Overview
On 4 July 2014, orphan designation (EU/3/14/1282) was granted by the European Commission to Fondazione Telethon, Italy, for mixture of two adeno-associated viral vectors serotype 8 containing the 5'-half sequence of human MYO7A gene and the 3'-half sequence of human MYO7A gene for the treatment of Usher syndrome.
For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.
Key facts
Active substance |
Mixture of two adeno-associated viral vectors serotype 8 containing the 5'-half sequence of human MYO7A gene and the 3'-half sequence of human MYO7A gene
|
Intended use |
Treatment of Usher syndrome
|
Orphan designation status |
Positive
|
EU designation number |
EU/3/14/1282
|
Date of designation |
04/07/2014
|
Sponsor |
Fondazione Telethon Ets
|
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
Update history
Date | Update |
---|---|
November 2022 | The sponsor's name was changed from Fondazione Telethon to Fondazione Telethon Ets in November 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: