EU/3/14/1306: Orphan designation for the treatment of Huntington’s disease

Cysteamine bitartrate (gastroresistant) (mercaptamine)


On 29 July 2014, orphan designation (EU/3/14/1306) was granted by the European Commission to Raptor Pharmaceuticals Europe BV, the Netherlands, for cysteamine bitartrate for the treatment of Huntington's disease.

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)
Intended use
Treatment of Huntington’s disease
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

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:

  • 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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