On 18 November 2016, orphan designation (EU/3/16/1769) was granted by the European Commission to SynteractHCR Deutschland GmbH, Germany, for 2-hydroxy-6-((2-(1-isopropyl-1H-pyrazol-5-yl)pyridin-3-yl)methoxy)benzaldehyde (also known as GBT440) for the treatment of sickle cell disease.
This medicine is now known as voxelotor.
The sponsorship was transferred to Global Blood Therapeutics Netherlands B.V., the Netherlands in January 2021.
Voxelotor has been authorised in the EU as Oxbryta since 14 February 2022.
Treatment of sickle cell disease
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Review of designation
Documents related to this orphan designation evaluation
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: