Also known as: Oxbryta
Voxelotor was marketed as OXBRYTA, a hemoglobin S polymerization inhibitor initially approved under accelerated approval for sickle cell disease. In September 2024, the manufacturer voluntarily withdrew Oxbryta from worldwide markets due to safety concerns, and FDA alerted patients and clinicians accordingly.
Approved gene-editing therapies for sickle cell disease priced at $2.2 million remain out of reach in Africa, where 80% of the world's cases occur, as Uganda launches mandatory newborn screening.
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| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT06023199 |
Assessing Physical Function in Sickle Cell Patients Taking Voxelotor |
ACTIVE_NOT_RECRUITING | PHASE2 |
| NCT05981365 |
Voxelotor CYP and Transporter Cocktail Interaction Study |
COMPLETED | PHASE1 |
| NCT05289570 |
Voxelotor for Improving Oxygen Saturation in Adults |
TERMINATED | PHASE2 |
| NCT05018728 |
The Effect of Voxelotor on Cerebral Hemodynamic Response in Children With Sickle Cell Anemia |
TERMINATED | PHASE2 |
| NCT04724421 |
Expanded Access Protocol for Adults and Pediatric Patients With Sickle Cell Disease Who Have No Alternative Treatment Options |
NO_LONGER_AVAILABLE | |
| NCT04581356 |
Voxelotor Sickle Cell Exercise Study |
COMPLETED | PHASE4 |
| NCT04400487 |
Actigraphy Improvement With Voxelotor (ActIVe) Study |
COMPLETED | PHASE4 |
| NCT04335721 |
A Voxelotor for Sickle Cell Anemia Patients at Highest Risk for Progression of Chronic Kidney Disease |
TERMINATED | PHASE1/PHASE2 |
| NCT04247594 |
Dose Escalation Study to Evaluate the Safety, Tolerability, PK and PD of Voxelotor in Patients With SCD |
TERMINATED | PHASE2 |
| NCT04218084 |
Study to Evaluate the Effect of GBT440 on TCD in Pediatrics With Sickle Cell Disease |
TERMINATED | PHASE3 |
