We still Hope
On November 25, 2019, the Food and Drug Administration granted accelerated approval to voxelotor (Oxbryta, Global Blood Therapeutics) for adults and pediatric patients 12 years of age and older with sickle cell disease.
The primary efficacy outcome measure was Hb response rate defined as an Hb increase of >1 g/dL from baseline to week 24. The response rate for voxelotor was 51.1% (46/90) compared to 6.5% (6/92) in the placebo group (p<0.0001). Additional efficacy evaluation included change in Hb, percent change in indirect bilirubin and percent reticulocyte count during this time period. In the voxelotor 1,500 mg group, the mean change for Hb, indirect bilirubin, and percent reticulocyte count were 1.14g/dL, ‑29.08%, and -19.93%, respectively. In the placebo group, the mean change during this time period for Hb, indirect bilirubin, and percent reticulocyte count were ‑0.08g/dL, ‑3.16%, and 4.54%, respectively.
Read the full press release from the FDA here: