EU/3/22/2598: Orphan designation for the treatment of giant axonal neuropathy

Adeno-associated virus vector serotype 9 encoding human gigaxonin gene

Table of contents


This medicine was designated as an orphan medicine for the treatment of giant axonal neuropathy in the European Union on 13 April 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
Adeno-associated virus vector serotype 9 encoding human gigaxonin gene
Intended use
Treatment of giant axonal neuropathy
Orphan designation status
EU designation number
Date of designation

Raremoon Consulting Esp S.L.
Ronda De Sant Pere 33 Ppal 3 Y 4
08010 Barcelona

  • List item

    Minutes of the COMP meeting on 15-17 March 2022 (PDF/505.22 KB)


    First published: 13/06/2022
    Last updated: 22/09/2022

  • 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:

    How useful was this page?

    Add your rating