EU/3/10/817

About

On 17 December 2010, orphan designation (EU/3/10/817) was granted by the European Commission to Genable Technologies Ltd, Ireland, for adeno-associated viral vector containing DNA encoding an RNAi targeting rhodopsin / adeno-associated viral vector containing a rhodopsin gene for the treatment of rhodopsin-linked retinitis pigmentosa.

In March 2017, Genable Technologies Ltd changed name to Spark Therapeutics Ireland Ltd.

Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in December 2018 on request of the Sponsor.

Key facts

Active substance
  • Adeno-associated viral vector containing DNA encoding an RNAi targeting rhodopsin
  • adeno-associated viral vector containing a rhodopsin gene
Disease / condition
Treatment of rhodopsin-linked retinitis pigmentosa
Date of first decision
17/12/2010
Outcome
Withdrawn
EU designation number
EU/3/10/817

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

Spark Therapeutics Ireland Ltd
The Tower
Trinity Technology & Enterprise Campus
Pearse Street
Dublin 2
Ireland
Tel:  +353 87 2865356
E-mail:  info@sparktx.com

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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