Oxbryta – pro
Oxbryta (voxelotor) tablets are for the treatment of sickle cell disease (SCD) in adults and children 12 years of age and older. The accelerated approval of Oxbryta is based on clinically meaningful and statistically significant improvements in hemoglobin levels, accompanied by reductions in red blood cell destruction (hemolysis). Data from the Phase 3 HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) Study of 274 patients 12 years of age and older with SCD showed that, after 24 weeks of treatment, 51.1% of patients receiving Oxbryta achieved a greater than 1 g/dL increase in hemoglobin compared with 6.5% receiving placebo (p<0.001). Results from the HOPE Study were published in June 2019 in The New England Journal of Medicine. The current understanding is that chronically low HB levels lead to end-organ damage.
E. Vichinsky et al, A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease. N Engl J Med 2019; 381:509-519