25 November 2019 - The Food and Drug Administration has granted accelerated approval to voxelotor (Oxbryta, Global Blood Therapeutics) for adults and paediatric patients 12 years of age and older with sickle cell disease.

Efficacy was evaluated in 274 patients with sickle cell disease in HOPE, a randomised, double-blind, placebo-controlled, multicenter trial. Patients were randomised to voxelotor 1500 mg (N=90), 900 mg (N=92), or placebo (N=92). The median age was 24 years (range 12, 64). Approximately 65% of patients were taking hydroxyurea at trial entry. Patients were enrolled if their baseline hemoglobin (Hb) ≥5.5 to ≤10.5 g/dL. Patients on stable hydroxyurea doses continued the drug throughout the trial. Randomisation was stratified by whether the patient was already receiving hydroxyurea, by geographic region, and by age.

This indication is approved under accelerated approval based on increase in haemoglobin. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s). FDA granted this application priority review, fast track, and breakthrough therapy designation.

Read FDA press release