EU/3/16/1789: Orphan designation for the treatment of sickle cell disease

synthetic human hepcidin

Table of contents


On 18 November 2016, orphan designation (EU/3/16/1789) was granted by the European Commission to EMAS Pharma Limited, United Kingdom, for synthetic human hepcidin for the treatment of sickle cell disease.

The sponsorship was transferred to Cogas Pharma Limited, Ireland, in July 2017 and subsequently to La Jolla Pharmaceutical II BV, Netherlands, in March 2018.

Please note that this product was withdrawn from the Union Register of orphan medicinal products in May 2022 on request of the Sponsor.

Key facts

Active substance
synthetic human hepcidin
Intended use
Treatment of sickle cell disease
Orphan designation status
EU designation number
Date of designation
La Jolla Pharmaceutical II BV
Herengracht 500
1017 CB Amsterdam
The Netherlands
Tel. +31 20 737 2218

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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