Regulatory and Pipeline Updates in Sickle Cell Disease from GBT

Global Blood Therapeutics, Inc. (GBT) announced updates across several of its development programs in sickle cell disease (SCD), on 22 of July. GBT introduced two global, randomized, placebo-controlled, pivotal Phase 3 clinical trials of inclacumab, a novel P-selectin inhibitor. Also enrolled the first SCD patient in a Phase 1 study evaluating GBT021601 (GBT601), a next-generation hemoglobin S (HbS) polymerization inhibitor, in people with SCD. Furthermore, the company has submitted a supplemental New Drug Application to the U.S. Food and Drug Administration seeking accelerated approval for Oxbryta® (voxelotor) for the treatment of SCD in children ages 4 to 11 years, together with a related separate New Drug Application (NDA) required to seek approval for a pediatric weight-based formulation of Oxbryta. President and CEO of GBT believes that the supplemental New Drug Application for Oxbryta creates a significant potential to impact the longer-term outcomes by addressing the root cause of red blood cell sickling at a young age. More info: Global Blood Therapeutics, Inc. press release.