EU/3/10/778: Orphan designation for the treatment of cystinosis

Cysteamine bitartrate (gastroresistant) (mercaptamine)


Cysteamine bitartrate (gastroresistant) has been authorised in the EU as Procysbi since 6 September 2013.

On 20 September 2010, orphan designation (EU/3/10/778) was granted by the European Commission to Raptor Pharmaceuticals Europe BV, the Netherlands, for cysteamine bitartrate (gastroresistant) for the treatment of cystinosis.

This medicine is now known as mercaptamine.

In February 2017, Raptor Pharmaceuticals Europe B.V changed name to Horizon Pharma Europe B.V.

In November 2017, Horizon Phama Europe B.V. changed name to Chiesi Orphan B.V.

The sponsorship was transferred to Chiesi Farmaceutici S.p.A., Italy, in May 2018.

Key facts

Active substance
Cysteamine bitartrate (gastroresistant) (mercaptamine)
Medicine name
Intended use
Treatment of cystinosis
Orphan designation status
EU designation number
Date of designation

Chiesi Farmaceutici S.p.A.
Via Palermo 26/A
43122 Parma
Tel. +39 0521 279 793

Review of designation

During its meeting of 9-11 July 2013, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/10/778 for Procysbi (mercaptamine1) 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 satisfactory methods of treatment. As other methods of treatment for patients with this condition are authorised in the European Union (EU), the COMP also looked at the significant benefit of the product over existing treatments. The COMP recommended that the orphan designation of the medicine be maintained2.

1Previously known as cysteamine bitartrate (gastroresistant).
2The 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: Procysbi (mercaptamine) for the treatment of cystinosis (PDF/80.4 KB)


    First published: 10/10/2013
    Last updated: 10/10/2013

  • 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:

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