Drug updated on 4/18/2024

Dosage FormTablet (oral; 300 mg and 500 mg), Tablet for oral suspension (oral; 300 mg)
Drug ClassHemoglobin S polymerization inhibitors
Ongoing and
Completed Studies


  • Indicated for the treatment of sickle cell disease in adults and pediatric patients 4 years of age and older.

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  • Voxelotor (Oxbryta) is indicated for the treatment of sickle cell disease in adults and pediatric patients 4 years of age and older. It has been noted to significantly increase hemoglobin levels, offering a new approach that addresses the root cause of this condition.
  • The information was derived from five systematic reviews/meta-analyses that compared voxelotor's safety and effectiveness with other treatments such as hydroxyurea, crizanlizumab, and L-glutamine, among others.
  • While voxelotor's impact on reducing vaso-occlusive crises (VOCs), a key complication of sickle cell disease, was not significant when compared to L-glutamine or crizanlizumab, its ability to increase hemoglobin levels presents a strong case for its use, especially in patients where VOCs are not the sole concern.
  • In terms of safety profile comparison with other treatments like L-glutamine and crizanlizumab, voxelotor is well-tolerated, but there exists theoretical concerns about potentially impaired oxygen delivery due to hemoglobin modification which requires further evaluation.
  • There seems to be potential benefit in combination therapy, particularly involving voxelotor plus L-glutamine, despite the less significant effect on VOC frequencies by voxelotor alone. This points towards the valuable place it holds within a comprehensive treatment strategy for sickle cell disease.
  • More research is needed regarding usage data across different demographic groups, including the Arabic population suffering from SCD, as highlighted in one study. Ongoing evaluations also exist concerning younger pediatric patients through the HOPE-KIDS trial, indicating the need for understanding effectiveness and safety across various genetic backgrounds.