EU/3/18/1973: Orphan designation for the treatment of retinitis pigmentosa

2'-O-(2-Methoxyethyl)-modified antisense oligonucleotide targeting exon 13 in the USH2A gene

Table of contents

Overview

On 22 February 2018, orphan designation (EU/3/18/1973) was granted by the European Commission to ProQR Therapeutics IV BV, the Netherlands, for 2'-O-(2-methoxyethyl)-modified antisense oligonucleotide targeting exon 13 in the USH2A gene (also known as QR 421a) for the treatment of retinitis pigmentosa.

Key facts

Active substance
2'-O-(2-Methoxyethyl)-modified antisense oligonucleotide targeting exon 13 in the USH2A gene
Intended use
Treatment of retinitis pigmentosa
Orphan designation status
Positive
EU designation number
EU/3/18/1973
Date of designation
22/02/2018
Sponsor
ProQR Therapeutics IV BV
Zernikedreef 9
2333 CK Leiden
The Netherlands
Tel. +31 88 166 7000
E-mail: info@proqr.com

Documents related to this orphan designation evaluation

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.

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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