EU/3/12/981

About

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.

Key facts

Active substance
Adenovirus-associated viral vector serotype 2 containing the human RPE65 gene (voretigene neparvovec)
Medicine name
Luxturna
Disease / condition
Treatment of leber's congenital amaurosis
Date of decision
02/04/2012
Outcome
Positive
Orphan decision number
EU/3/12/981

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 iconorphan medicine assessment report

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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