EU/3/22/2636: Orphan designation for the treatment of Angelman syndrome

2'-O-(2-methoxyethyl) modified antisense oligonucleotide targeting UBE3A antisense transcript RNA

Table of contents

Overview

This medicine was designated as an orphan medicine for the Treatment of Angelman syndrome in the European Union on 21 June 2022.

This means that the developer will receive scientific and regulatory support from EMA to advance their medicine to the stage where they can apply for a marketing authorisation.

Orphan designation does not mean the medicine is available or authorised for use. All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.

During the medicine's development, doctors may be able to enrol patients in clinical trials investigating the medicine. For information on ongoing clinical trials in the EU, see:

Key facts

Active substance
2'-O-(2-methoxyethyl) modified antisense oligonucleotide targeting UBE3A antisense transcript RNA
Intended use
Treatment of Angelman syndrome
Orphan designation status
Positive
EU designation number
EU/3/22/2636
Date of designation
21/06/2022
Sponsor

Ionis Development (Ireland) Limited
10 Earlsfort Terrace
Dublin 2 D02 T380
Co. Dublin
Ireland
Tel. + 8006794747
E-mail: patients@ionisph.com

  • List item

    Minutes of the COMP meeting on 10-12 May 2022 (PDF/517.18 KB)


    First published: 21/07/2022
    EMA/COMP/260156/2022

  • 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.

    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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