TABLE 1.
Approved and potential investigational therapeutics for hemophilia management.
| Category/[citations] | Generic name | Brand name | Manufacturer | Indication a | Clinical stage | Approval b |
|---|---|---|---|---|---|---|
| Plasma-derived concentrates (PDCs) | ||||||
| PDC-FVIII + VWF (Kouides et al., 2017; Boban et al., 2024; Federici et al., 2024) | Antihemophilic factor/VWF complex (human) | Humate-P Haemate P | CSL Behring | Prophylaxis and treatment for HA (adult) and VWD (adult and pediatric) | FDA, EMA | |
| Antihemophilic factor/VWF complex (human) | Wilate | Octapharma | Prophylaxis and treatment for HA (age ≥12 years) and mild/moderate VWD (age ≥6 years) | FDA, EMA | ||
| Antihemophilic factor/VWF complex (human) | Alphanate | Grifols | Prophylaxis and treatment for HA and VWD (adult and pediatric) | FDA | ||
| PDC-FVIII (Elalfy et al., 2022) | Antihemophilic factor (human) | Koate-DVI | Kedrion | Prophylaxis and treatment for HA (adult and pediatric) | FDA | |
| PDC-FIX (Hoots et al., 2003; Quon and Logan, 2011; Serban et al., 2012) | Coagulation factor IX (human) | Mononine | CSL Behring | Prophylaxis and treatment for HB (adult and pediatric) | FDA, EMA | |
| Coagulation factor IX (human) | AlphaNine | Grifols | Prophylaxis and treatment for HB (adult and pediatric) | FDA | ||
| Coagulation factor IX (human) | Haemonine | DHZ Pharma | Prophylaxis and treatment for HB (age ≥6 years) | EMA | ||
| Recombinant coagulation factors | ||||||
| rFVIII (Shapiro, 2007; Turecek et al., 2016; Chowdary et al., 2016; St. Ledger et al., 2018) | Octocog alfa; Antihemophilic factor (recombinant) | Advate | Takeda | Prophylaxis and treatment for HA (adult and pediatric) | FDA, EMA | |
| Antihemophilic factor (recombinant), PEGylated | Adynovate | Takeda | Prophylaxis and treatment for HA (adult and pediatric) | FDA, EMA | ||
| Antihemophilic factor (recombinant), Fc fusion protein | Eloctate | Biogen Idec | Prophylaxis and treatment for HA (adult and pediatric) | FDA, EMA | ||
| Lonoctocog alfa; Antihemophilic factor (recombinant), single chain | Afstyla | CSL Behring | Prophylaxis and treatment for HA (adult and pediatric) | FDA, EMA | ||
| rFIX (Lambert et al., 2007; Ezban et al., 2019; Lyseng-Williamson, 2017) | Nonacog alfa; Coagulation factor IX (recombinant) | BeneFIX | Pfizer | Prophylaxis and treatment for HB (adult and pediatric) | FDA, EMA | |
| Coagulation factor IX (recombinant), glycoPEGylated | Rebinyn | Novo Nordisk | Prophylaxis and treatment for HB (adult and pediatric) | FDA, EMA | ||
| Albutrepenonacog alfa: Coagulation factor IX (recombinant), albumin fusion protein | Idelvion | CSL Behring | Prophylaxis and treatment for HB (adult and pediatric) | FDA, EMA | ||
| Bypassing agents | ||||||
| aPCC (Ettingshausen et al., 2023) | Anti-inhibitor coagulant complex | FEIBA | Takeda | Prophylaxis and treatment for HA & HB with inhibitor (adult and pediatric) | FDA, EMA | |
| rFVIIa (Shima, 2024) | Coagulation factor VIIa (recombinant) | NovoSeven | Novo Nordisk | Prophylaxis and treatment for HA & HB with inhibitor (adult and pediatric) | FDA, EMA | |
| Non-factor therapeutics | ||||||
| Bispecific antibody (Shapiro et al., 2018) | Emicizumab | Hemlibra | Roche/Genentech | Prophylaxis rather than acute treatment for HA (adult and pediatric) via subcutaneous route | FDA, EMA | |
| Anti-TFPI antibody (Lamb, 2025; Keam, 2023; Mancuso et al., 2022) | Concizumab | Alhemo | Novo Nordisk | Prophylaxis for HA & HB (age ≥12 years) via subcutaneous route | FDA, EMA | |
| Marstacimab | Hympavzi | Pfizer | Prophylaxis for HA & HB (age ≥12 years) via subcutaneous route, specifically without inhibitors | FDA, EMA | ||
| Befovacimab (BAY 1093884) | Bayer | Treatment for severe HA & HB via subcutaneous route | Phase 2 | |||
| KN057 | Suzhou Alphamab | Prophylaxis for moderate to severe HA & HB via subcutaneous route | Phase 3 | |||
| MG1113 | GC Biopharma | Severe HA & HB via subcutaneous route | Phase 1/1b | |||
| siRNA [66–68] | Fitusiran; GalNAc-conjugated siRNA targeting AT mRNA | Qfitlia | Sanofi | Prophylaxis for HA & HB (aged ≥12 years) via subcutaneous route | Phase 3 (For age ≤12 years) | FDA |
| Viral vector-based Gene therapy | ||||||
| AAV5-FIX vector (Heo, 2023; Sekayan et al., 2023; Anguela and High, 2024; von Drygalski et al., 2025) | Etranacogene dezaparvovec | Hemgenix | uniQure | One-time treatment for moderate/severe HB (age ≥18 years) | FDA, EMA | |
| AAV5-FVIII vector (Blair, 2022; Ozelo et al., 2022; Mahlangu et al., 2023; Samelson-Jones et al., 2024; Symington et al., 2024) | Valoctocogene roxaparvovec | Roctavian | BioMarin | One-time treatment for severe HA without inhibitors to AAV5 and FVIII (age ≥18 years) | FDA, EMA | |
| AAVRh74-FIX vector (Cuker et al., 2024) | Fidanacogene elaparvovec | Beqvez (Production end in 2025) | Pfizer | One-time treatment for moderate/severe HB without inhibitors to AAVRh74 and FIX (age ≥18 years) | FDA | |
| Autologous stem cells | ||||||
| Autologous HSCs (Doering et al., 2018; Srivastava et al., 2025) | CD68-ET3-LV CD34+ | Severe HA | Early phase 1 | |||
a
Unless otherwise specified, all drugs were administered intravenously.
b
The approval shows only by FDA, and European Medicines Agency (EMA).