On 23 August 2017, orphan designation (EU/3/17/1899) was granted by the European Commission to ProQR Therapeutics IV BV, the Netherlands, for antisense oligonucleotide targeting exon 13 in the USH2A gene (also known as QR-421) for the treatment of retinitis pigmentosa.

Key facts

Active substance
Antisense oligonucleotide targeting exon 13 in the USH2A gene
Medicine name
Disease / condition
Treatment of retinitis pigmentosa
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

ProQR Therapeutics IV BV
Zernikedreef 9
2333 CK Leiden
The Netherlands
Tel. +31 653 719 770

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.

  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

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