EU/3/07/434

About

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
idebenone
Disease / condition
Treatment of Leber's hereditary optic neuropathy
Date of first decision
16/02/2007
Outcome
Positive
EU designation number
EU/3/07/434

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.

Sponsor's contact details

Santhera Pharmaceuticals (Deutschland) GmbH
Marie-Curie Strasse 8
D-79539 Lörrach
Germany
Tel. +49 7621 1690200
Fax +49 7621 1690201
E-mail: office@santhera.com

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.

How useful was this page?

Add your rating