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Data Support Voxelotor as a Potential Disease-Modifying Therapy for Sickle Cell Disease

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An ongoing study assessing the safety, pharmacokinetics, and efficacy of voxelotor as a potential disease-modifying therapy in pediatric patients with sickle cell disease (SCD) has shown promising preliminary results. Voxelotor is an oral therapy that modulates hemoglobin’s oxygen affinity, inhibiting hemoglobin polymerization.

The study is being conducted in two parts: part A, which is assessing a single dose of voxelotor 600 mg in pediatric and adolescent patients; and part B, which is assessing multiple doses of voxelotor 900 mg/d or 1500 mg/d for 24 weeks in adolescents. As of November 6, 2017, 24 patients had received voxelotor 900 mg and 12 patients had received voxelotor for ≥16 weeks. Of those receiving voxelotor for ≥16 weeks, data for hemolysis measures were available for 11 patients. Six patients (55%) achieved a hemoglobin response of >1 g/dL increase and had concordant improvements in laboratory markers of hemolysis, with median reductions in reticulocytes and indirect bilirubin of 11% and 40%, respectively. Among the 12 patients treated for ≥16 weeks, 10 (83%) had reduction in total symptom scores (TSS) at week 16, with a 94% median reduction in TSS from baseline. The treatment was well-tolerated, with no serious adverse events (AEs) or drug discontinuations due to AEs.

Read more here.


Hoppe CC, Inati AC, Brown C, et al. Initial results from a cohort in a phase 2a study (GBT440-007) evaluating adolescents with sickle cell disease treated with multiple doses of voxelotor, a sickle hemoglobin polymerization inhibitor. Presented at: 2018 American Society of Pediatric Hematology/Oncology; May 2-5, 2018; Pittsburgh, Pennsylvania. Abstract 301.

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