EU/3/07/434: Orphan designation for the treatment of Leber's hereditary optic neuropathy



On 15 February 2007, orphan designation (EU/3/07/434) was granted by the European Commission to Santhera Pharmaceuticals (Deutschland) GmbH, Germany, for idebenone for the treatment of Leber's hereditary optic neuropathy.

The name of the sponsor changed to Santhera Pharmaceuticals (Deutschland) GmbH in September 2010.

Update: idebenone (Raxone) has been authorised in the EU since 8 September 2015 for treatment of visual impairment in adolescent and adult patients with Leber's Hereditary Optic Neuropathy (LHON).

More information on Raxone can be found in the European public assessment report (EPAR).

Key facts

Active substance
Intended use
Treatment of Leber's hereditary optic neuropathy
Orphan designation status
EU designation number
Date of designation

Santhera Pharmaceuticals (Deutschland) GmbH
Marie-Curie Strasse 8
D-79539 Lörrach
Tel. +49 7621 1690200
Fax +49 7621 1690201

Review of designation

During its meeting of 14 to 16 July 2015, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/07/434 for Raxone (idebenone) as an orphan medicinal product for the treatment of Leber's hereditary optic neuropathy. The COMP assessed whether, at the time of marketing authorisation, the medicinal product still met the criteria for orphan designation. The Committee looked at the seriousness and prevalence of the condition, and the existence of other methods of treatment. The COMP recommended that the orphan designation of the medicine be maintained1.

1The maintenance of the orphan designation at time of marketing authorisation would, except in specific situations, give an orphan medicinal product 10 years of market exclusivity in the EU. This means that in the 10 years after its authorisation similar products with a comparable therapeutic indication cannot be placed on the market.

Documents related to this orphan designation evaluation

  • List item

    Recommendation for maintenance of orphan designation at the time of marketing authorisation: Raxone (idebenone) for the treatment of Leber’s hereditary optic neuropathy (PDF/78.59 KB)


    First published: 22/09/2015
    Last updated: 22/09/2015

  • 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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