Products supporting long-term prophylaxis that are enhanced over standard half-life factor products
| 1. Extended half-life recombinant factors supporting once-weekly administration |
| FIX |
| a. Alprolix, coagulation FIX (recombinant), Fc fusion protein, efmoroctocog alfa, Biogen Sanofi |
| b. Idelvion, coagulation FIX (recombinant), albutrepenonacog alfa, albumin fusion protein, CSL Behring |
| c. Rebinyn, coagulation FIX (recombinant), glycopegylated, nonacog beta pegol, Novo Nordisk |
| FVIII |
| d. Efanesoctocog alfa (BIVV001, rFVIIIc-VWF-XTEN), in clinical trials, Sobi and Sanofi |
| 2. FVIII mimetics (bivalent monoclonal antibodies that promote activation of FX in the absence of FVIII) |
| a. Hemlibra, emicizumab, Genentech, Roche |
| b. Mim8, Novo Nordisk, in clinical trials |
| 3. Hemostatic rebalancing agents |
| a. Antithrombin siRNA, fitusiran, Sanofi, in clinical trials |
| b. TFPI monoclonal antibodies |
| i. Concizumab, humanized immunoglobulin G4 in clinical trials, Novo Nordisk |
| ii. Marstacimab, humanized immunoglobulin G1 in clinical trials, Pfizer |
| c. Lowering of protein C or protein S |
| i. Discussed, not yet in clinical trials |
| 4. Gene therapy |
| FIX |
| a. Fidanacogene elaparvovec, SPK 9001, in clinical trials, Pfizer |
| b. Etranacogene dezaparvovec, AMT-061, in clinical trials, uniQure |
| c. scAAV2/8-LP1-hFIXcoAAV8, in clinical trials, St. Jude, University College London, and the Royal Free Hospital |
| FVIII |
| d. Valoctocogene roxaparvovec, 270-303, in clinical trials, BioMarin |
| e. SPK-8011, in clinical trials, Spark Therapeutics |
| 1. Extended half-life recombinant factors supporting once-weekly administration |
| FIX |
| a. Alprolix, coagulation FIX (recombinant), Fc fusion protein, efmoroctocog alfa, Biogen Sanofi |
| b. Idelvion, coagulation FIX (recombinant), albutrepenonacog alfa, albumin fusion protein, CSL Behring |
| c. Rebinyn, coagulation FIX (recombinant), glycopegylated, nonacog beta pegol, Novo Nordisk |
| FVIII |
| d. Efanesoctocog alfa (BIVV001, rFVIIIc-VWF-XTEN), in clinical trials, Sobi and Sanofi |
| 2. FVIII mimetics (bivalent monoclonal antibodies that promote activation of FX in the absence of FVIII) |
| a. Hemlibra, emicizumab, Genentech, Roche |
| b. Mim8, Novo Nordisk, in clinical trials |
| 3. Hemostatic rebalancing agents |
| a. Antithrombin siRNA, fitusiran, Sanofi, in clinical trials |
| b. TFPI monoclonal antibodies |
| i. Concizumab, humanized immunoglobulin G4 in clinical trials, Novo Nordisk |
| ii. Marstacimab, humanized immunoglobulin G1 in clinical trials, Pfizer |
| c. Lowering of protein C or protein S |
| i. Discussed, not yet in clinical trials |
| 4. Gene therapy |
| FIX |
| a. Fidanacogene elaparvovec, SPK 9001, in clinical trials, Pfizer |
| b. Etranacogene dezaparvovec, AMT-061, in clinical trials, uniQure |
| c. scAAV2/8-LP1-hFIXcoAAV8, in clinical trials, St. Jude, University College London, and the Royal Free Hospital |
| FVIII |
| d. Valoctocogene roxaparvovec, 270-303, in clinical trials, BioMarin |
| e. SPK-8011, in clinical trials, Spark Therapeutics |