Voxelotor: A Novel Treatment for Sickle Cell Disease.

The Annals of Pharmacotherapy
Leah B HerityDenise Kozella Lowe

Abstract

To review the pharmacological characteristics, clinical evidence, and place in therapy of voxelotor for the treatment of sickle cell disease (SCD). A comprehensive literature search of PubMed (1966 to April 2020) was conducted. Key search terms included GBT440, sickle cell, and voxelotor. Other sources were derived from bibliographies of articles, product labeling, manufacturer's website, and news releases. ClinicalTrials.gov was searched for additional studies. All English-language articles identified from the data sources were reviewed and evaluated. Case reports/series and phase 1 through 3 clinical trials were included. SCD is an inherited disorder associated with significant morbidity and early mortality. Three medications approved for SCD reduce SCD-associated complications but do not selectively ameliorate the underlying disease. Voxelotor is a novel agent that targets the pathophysiology of SCD. A phase 3 trial reported an increase in mean Hb level from baseline for voxelotor compared with placebo (1.1 vs -0.1 g/dL; P < 0.001). Voxelotor is generally well tolerated, with common adverse effects including headache, diarrhea, nausea, and arthralgia. Voxelotor may be considered for patients with SCD who have continued anemi...Continue Reading

References

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Jun 15, 2019·The New England Journal of Medicine·Elliott VichinskyUNKNOWN HOPE Trial Investigators

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Citations

Sep 4, 2021·American Journal of Hematology·Matthew LeeSusanna A Curtis

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Clinical Trials Mentioned

NCT02285088
NCT03041909
NCT03036813
NCT02850406
NCT04218084
NCT04188509
NCT04247594
NCT03573882

Software Mentioned

voxelotor

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