On Nov 25, the U.S. Food and Drug Administration granted accelerated approval to Oxbryta (voxelotor) for the treatment of sickle cell disease (SCD) in adults and pediatric patients 12 years of age and older.
| Unmet Medical Need Parameters | Unmet Medical Need in sickle cell disease after SIKLOS | Unmet Medical Need in sickle cell disease after OXBRYTA |
|---|---|---|
| Treatment Adequacy | Current treatment increases hemoglobin F levels in red blood cells (RBCs), decreasing neutrophils, increasing the water content of RBCs, increasing deformability of sickled cells, and altering the adhesion of RBCs to endothelium. | New treatment increases the affinity of Hb for oxygen, inhibition of HbS polymerization, inhibit RBC sickling, improve RBC deformability, and reduce whole blood viscosity. |
| Disease Severity | • Vaso-occlusive crisis and chronic pain are associated with considerable economic loss and disability • Approved in pediatric patients, 2 years of age and older | • Vaso-occlusive crisis and chronic pain are associated with considerable economic loss and disability • Approved in adults and pediatric patients 12 years of age and older. |
| Concerns of Payers & Providers | • Hemoglobin F percentage change • Vasoocclusive episodes frequency • Acute chest syndrome frequency • Hospitalization rate related to SCD • Blood transfusion need | • Hemoglobin F percentage change |
| Net Unmet Medical Need Score | 2.46/5 | 2.50/5 |
| Low to moderate unmet medical need | Low to moderate unmet medical need |
OXBRYTA has disease modifying effect, increases the affinity of Hb for oxygen, inhibition of HbS polymerization, inhibit RBC sickling, improve RBC deformability, and reduce whole blood viscosity.
To get detailed analysis of unmet medical need and to understand our approach which we are using for unmet medical analysis of any disease please contact at [email protected]
We help researchers in unmet medical need analysis of diseases which helps them in disease prioritization for any new drug/device/target/MOA development. We do unmet medical need analysis of any disease based on various parameters which is not limited to treatment adequacy, patient perspective, payer & providers perspective.
Leave a Reply