EU/3/12/981: Orphan designation for the treatment of Leber's congenital amaurosis

Adenovirus-associated viral vector serotype 2 containing the human RPE65 gene (voretigene neparvovec)

Overview

This medicine is now known as voretigene neparvovec.

On 2 April 2012, orphan designation (EU/3/12/981) was granted by the European Commission to Alan Boyd Consultants Ltd, United Kingdom, for adenovirus-associated viral vector serotype 2 containing the human RPE65 gene for the treatment of Leber's congenital amaurosis.

The sponsorship was transferred to Spark Therapeutics Ireland Ltd, in March 2017.

Adenovirus-associated viral vector serotype 2 containing the human RPE65 gene in the amended indication treatment of inherited retinal dystrophies has been authorised in the EU as Luxturna since 22 November 2018.

The sponsorship was transferred to Novartis Europharm Limited, Ireland, in February 2019.

Key facts

Active substance
Adenovirus-associated viral vector serotype 2 containing the human RPE65 gene (voretigene neparvovec)
Medicine name
Luxturna
Intended use
Treatment of Leber's congenital amaurosis
Orphan designation status
Positive
EU designation number
EU/3/12/981
Date of designation
02/04/2012
Sponsor

Novartis Europharm Limited
Vista Building
Elm Park, Merrion Road
Dublin 4
Ireland
Tel.: +41 (0)61 324 11 11
E-mail: orphan.enquiries@novartis.com

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of Luxturna at the time of marketing authorisation, and confirmed that the orphan designation should be maintained.

More information is available in the  PDF icon orphan medicine assessment report

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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