Comparison with nonfactor products
| . | Emicizumab . | Fitusiran . | Concizumab . |
|---|---|---|---|
| Manufacturer | Chugai/Roche | Alnylam/Sanoffi | Novo Nordisk |
| Action mechanism | Bispecific antibody to FIXa/FIX and FX/FXa | siRNA targeting antithrombin | Anti-TFPI antibody |
| Administration | SC | SC | SC |
| t1/2 | 28.3-34.4 d | Shorter (plasma), longer (liver) | 74.8-114 h |
| Frequency | QW, Q2W, Q4W | QW, QM | Twice weekly, QD |
| Target patients | Hemophilia A noninhibitor, inhibitor | Hemophilia A, B noninhibitor, inhibitor | Hemophilia A, B noninhibitor, inhibitor |
| Risk of ADA | Positive: 1 case (HAVEN 2) and 3 cases (2; SQ, 1; IV, single injection for healthy volunteers) | None | Possible |
| Complication | TMA (n = 3), TE (n = 2) | TE (n = 1) | No reported |
| Other | Fatalities with compassionate use/expanded access (unrelated to drug) | ||
| Monitoring | aPTT, chromogenic | Antithrombin level | Free TFPI level |
| clot waveform | TGT | TGT | |
| ROTEM/TEG, TGT | |||
| Ongoing study | Phase 3: noninhibitor | Phase 3 | Phase 2 |
| QW, Q2W, Q4W | QW, QM | daily | |
| Phase 3: inhibitor | |||
| Q2W, Q4W | |||
| Approval | HEMLIBRA (inhibitor) | None | None |
| . | Emicizumab . | Fitusiran . | Concizumab . |
|---|---|---|---|
| Manufacturer | Chugai/Roche | Alnylam/Sanoffi | Novo Nordisk |
| Action mechanism | Bispecific antibody to FIXa/FIX and FX/FXa | siRNA targeting antithrombin | Anti-TFPI antibody |
| Administration | SC | SC | SC |
| t1/2 | 28.3-34.4 d | Shorter (plasma), longer (liver) | 74.8-114 h |
| Frequency | QW, Q2W, Q4W | QW, QM | Twice weekly, QD |
| Target patients | Hemophilia A noninhibitor, inhibitor | Hemophilia A, B noninhibitor, inhibitor | Hemophilia A, B noninhibitor, inhibitor |
| Risk of ADA | Positive: 1 case (HAVEN 2) and 3 cases (2; SQ, 1; IV, single injection for healthy volunteers) | None | Possible |
| Complication | TMA (n = 3), TE (n = 2) | TE (n = 1) | No reported |
| Other | Fatalities with compassionate use/expanded access (unrelated to drug) | ||
| Monitoring | aPTT, chromogenic | Antithrombin level | Free TFPI level |
| clot waveform | TGT | TGT | |
| ROTEM/TEG, TGT | |||
| Ongoing study | Phase 3: noninhibitor | Phase 3 | Phase 2 |
| QW, Q2W, Q4W | QW, QM | daily | |
| Phase 3: inhibitor | |||
| Q2W, Q4W | |||
| Approval | HEMLIBRA (inhibitor) | None | None |
QW, once weekly; Q2W, every 2 weeks; Q4W, monthly; SC, subcutaneous, siRNA, small interfering RNA; TGT, thrombin generation test.