The sponsorship was transferred to IDEA Innovative Drug European Associates (Ireland) Limited, Ireland, in September 2019.

On 12 October 2017, orphan designation (EU/3/17/1938) was granted by the European Commission to ProQR Therapeutics VII BV, the Netherlands, for antisense oligonucleotide targeting exon 73 in the COL7A1 gene (also known as QR-313) for the treatment of epidermolysis bullosa.

Key facts

Active substance
antisense oligonucleotide targeting exon 73 in the COL7A1 gene
Disease / condition
Treatment of epidermolysis bullosa
Date of first decision
EU designation number

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

Dundrum Business Park
Classon House 13
Dundrum Road
Dublin 14
Co. Dublin
D14 X9F9
Tel: +35319036117

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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