Stage (next event)
Phase 3 post-approval confirmatory study Readout
December 5-8, 2021
Catalyst Info & Data Links
TITLE: Voxelotor for Transcranial droppler (TCD) in SCD post-approval confirmatory study Readout
Clinical Trial (NCT03036813) Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE)
WHAT IS THE NEXT CATALYST EVENT?
post-approval confirmatory study Readout
WHEN WILL THE EVENT (OR DID THE EVENT) OCCUR?
December 5-8, 2020
MECHANISM OF ACTION
Oxbryta (voxelotor) is an oral, once-daily therapy for patients with sickle cell disease (SCD). Oxbryta works by increasing hemoglobin’s affinity for oxygen. Since oxygenated sickle hemoglobin does not polymerize, Oxbryta inhibits sickle hemoglobin polymerization and the resultant sickling and destruction of red blood cells. Through addressing hemolytic anemia and improving oxygen delivery throughout the body, GBT believes that Oxbryta has the potential to modify the course of SCD. On November 25, 2019, Oxbryta received U.S. Food and Drug Administration (FDA) accelerated approval for the treatment of SCD in adults and children 12 years of age and older.9 As a condition of accelerated approval, GBT will continue to study voxelotor in the HOPE-KIDS 2 Study, a post-approval confirmatory study using transcranial Doppler (TCD) flow velocity to assess the ability of Oxbryta to decrease stroke risk in children 2 to 15 years of age.
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