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Voxelotor is a hemoglobin oxygen-affinity modulator that is used to treat sickle cell disease. This study, published in Annals of Pharmacotherapy, conducted a comprehensive literature review on the safety, efficacy, and affordability of voxelotor.

Ultimately, it was found that voxelotor is generally well-tolerated and significantly increases mean hemoglobin level compared to placebo. Commonly noted adverse effects of volextor included arthralgia, diarrhea, headache, and nausea. Moreover, voxelotor may be considered for patients with sickle cell disease if they are hydroxyurea intolerant or have continued anemia and hemolysis despite being on a maximum tolerated dose of hydroxyurea.

Unfortunately, despite voxelotor’s favorable safety and efficacy profile and indication for the treatment of sickle cell disease, not all patients with the disease are able to reap its benefits secondary to cost.

The researchers concluded that voxelotor is a safe and effective monotherapy and combination therapy when used with hydroxyurea for the treatment of sickle cell disease in patients 12 and older. A call to action is made for providers to consider voxelotor’s cost and benefits before prescribing it for their patients [1].


[1] Herity, L. B., Vaughan, D. M., Rodriguez, L. R., & Lowe, D. K. (2020). Voxelotor: A novel treatment for sickle cell disease. Annals of Pharmacotherapy, 55(2), 240–245. https://doi.org/10.1177/1060028020943059

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