Update: 01 February 2019

The preliminary results from phase IIa HOPE trials for the safety and efficacy of voxelotor in SCD were presented at the 2018 ASH Annual Meeting. Results reflected the findings of two randomized, multi-centred trials with 154 patients participating – one with adults and adolescents over 12 years of age, and one with children and adolescents between the ages of 6 – 17.

Voxelotor is once-daily therapy for patients with SCD, is designed to work by helping hemoglobin, hold onto more oxygen as the red blood cells travel through the body. This keeps red blood cells in their normal shape and helps stop sickling. Thus leading to fewer VOCs.


  • Increases haemoglobin in a dose-dependent manner
  • 55% of patients experienced an Hb increase >1 g/dL after 16 weeks of treatment with Voxelotor 900 mg
  • Rapid, robust, and sustained improvement in haemoglobin and haemolysis
  • Safe and well tolerated
  • Fewer VOC with substantial increase in haemoglobin

The results also are potentially confounded by the concomitant administration of hydroxyurea.

However the results support a potential for voxelotor to reduce stroke risk in children – thus needing further investigation.

The FDA has granted Voxelotor Fast Track, Orphan Drug and Rare Pediatric Disease status. The EMA has designated Voxelotor as an Orphan Medicinal Product and included it in its Priority Medicines (PRIME) program.




https://www.ashclinicalnews.org/on-location/voxelotor-offers-new-hope-sickle-cell-disease/ https://www.gbt.com/file.cfm/22/docs/Interim-Results_Phase-2a-Study_Adolescents_final-presentation.pdf