Accelerated approval of Oxbryta ® (voxelotor): A case study on novel endpoint selection in sickle cell disease

Sickle cell disease (SCD) is an inherited disease characterized by hemolysis, anemia, and vaso-occlusion leading to substantial morbidity and mortality. Development of prior pharmacologic therapies exclusively utilized vaso-occlusive crisis (VOC) as a clinical efficacy endpoint; however, this focus on VOC did not capture the full extent of disease symptomatology and complications and slowed the development of new therapies. Voxelotor, a hemoglobin S polymerization inhibitor, was recently approved in the United States for the treatment of SCD in adults and adolescents 12  years of age and older through an accelerated approval pathway.
Source: Contemporary Clinical Trials - Category: Radiology Authors: Source Type: research