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

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
Via Varese 16 B
00185 Rome
RM
Italy
E-mail: regulatory@telethon.it

 

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.

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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