Voxelotor: First Approval

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Funding The preparation of this review was not supported by any external funding.

Conflict of interest During the peer review process the manufacturer of the agent under review was offered an opportunity to comment on the article. Changes resulting from any comments received were made by the authors on the basis of scientific completeness and accuracy. Hannah A. Blair is a salaried employee of Adis International Ltd/Springer Nature, is responsible for the article content and declares no relevant conflicts of interest.

Additional information about this Adis Drug Review can be found here.

Abstract Voxelotor (Oxbryta^™) is a haemoglobin S polymerization inhibitor that has been developed for the treatment of sickle cell disease. In November 2019, voxelotor received its first global approval in the USA for the treatment of sickle cell disease in adults and paediatric patients aged ≥ 12 years. The drug was granted accelerated approval based on the results of the phase III HOPE trial. Phase III clinical development of voxelotor for sickle cell disease is ongoing worldwide. Voxelotor also has Orphan Drug designation and Priority Medicine status in Europe for the treatment of sickle cell disease. This article summarizes the milestones in the development of voxelotor leading to this first approval for sickle cell disease.

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