Table 4.
Drug/treatment | MOA | USD |
---|---|---|
Hydroxyurea | Increase HbF | 16,800 |
L-glutamine | Decreased oxidation | 36,000 |
Crizanlizumab | P-seletin binding | 100,000 |
Voxelotor | Decreased hemolysis | 125,000 |
RBC exchange | Non-SS RBCs | 3,000* |
Allo-transplant | Corrected source | 145,000^ |
Gene therapy | BCL11A disruption | 2,700,000^ |
Gene therapy | CRISPR correct globin | >2,000,000^ |
This is cost per event not per year, RBC exchange may be monthly.
This is one time only cost.