EU/3/14/1341: Orphan designation for the treatment of cystinosis

Cysteamine hydrochloride


On 15 October 2014, orphan designation (EU/3/14/1341) was granted by the European Commission to Lucane Pharma SA, France, for cysteamine hydrochloride for the treatment of cystinosis.

Key facts

Active substance
Cysteamine hydrochloride
Medicine name
Intended use
Treatment of cystinosis
Orphan designation status
EU designation number
Date of designation
Lucane Pharma SA
172 rue de Charonne
75011 Paris
Tel. +33 1 53 86 87 50
Fax +33 1 43 71 14 96

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

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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