EU/3/08/578: Orphan designation for the treatment of cystinosis

Cysteamine hydrochloride


On 7 November 2008, orphan designation (EU/3/08/578) was granted by the European Commission to Orphan Europe SARL, France, for cysteamine hydrochloride for the treatment of cystinosis.

Cysteamine hydrochloride has been authorised in the EU as Cystadrops since 19 January 2017.

Key facts

Active substance
Cysteamine hydrochloride
Medicine name
Intended use
Treatment of cystinosis
Orphan designation status
EU designation number
Date of designation
Orphan Europe SARL
Immeuble Le Wilson
70 Avenue du Général de Gaulle
F-92800 Puteaux
Tel. +33 1 4773 6458
Fax +33 1 49 00 18 00

Review of designation

During its meeting of 6 to 8 December 2016, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/08/578 for Cystadrops (mercaptamine, previously known as cysteamine hydrochloride) as an orphan medicinal product for the treatment of cystinosis. 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. As other methods of treatment are authorised in the European Union (EU), the COMP also considered whether the medicine is of significant benefit to patients with cystinosis. 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 the same 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: Cystadrops (mercaptamine) for the treatment of cystinosis (PDF/80.37 KB)


    First published: 22/02/2017
    Last updated: 22/02/2017

  • 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:

    Related content

    How useful was this page?

    Add your rating