EU/3/17/1950

About

On 12 December 2017, orphan designation (EU/3/17/1950) was granted by the European Commission to MeiraGTx UK II Limited, United Kingdom, for adenovirus associated viral vector serotype 8 containing the human AIPL1 gene for the treatment of Leber's congenital amaurosis.

The sponsorship was transferred to MeiraGTx B.V., The Netherlands in May 2019.

Key facts

Active substance
Adenovirus associated viral vector serotype 8 containing the human AIPL1 gene
Disease / condition
Treatment of leber's congenital amaurosis
Date of first decision
12/12/2017
Outcome
Positive
EU designation number
EU/3/17/1950

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

MeiraGTx B.V.
Robert Boyleweg 4
2333 CG Leiden
Zuid-Holland
The Netherlands
Tel. +31 (0) 6480 67303
E-mail: info@meiragtx.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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