EU/3/16/1769: Orphan designation for the treatment of sickle cell disease

2-hydroxy-6-((2-(1-isopropyl-1H-pyrazol-5-yl)pyridin-3-yl)methoxy)benzaldehyde (voxelotor)


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.

Key facts

Active substance
2-hydroxy-6-((2-(1-isopropyl-1H-pyrazol-5-yl)pyridin-3-yl)methoxy)benzaldehyde (voxelotor)
Intended use
Treatment of sickle cell disease
Orphan designation status
EU designation number
Date of designation

Pfizer Europe MA EEIG
Boulevard De La Plaine 17
1050 Brussels
Brussels-Capital Region

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of Oxbryta at the time of marketing authorisation, and confirmed that the orphan designation should be maintained.

More information is available in the PDF icon orphan medicine assessment report .

Update history

May 2023The sponsorship was transferred from Global Blood Therapeutics Netherlands B.V., Netherlands to Pfizer Europe MA EEIG., Belgium.

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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