Table 2.
FDA-approved drugs for hepatocellular carcinoma.
| Drug | Classification | Target | Approval | Treatment | Clinical trial | Efficacy | Reference |
|---|---|---|---|---|---|---|---|
| Sorafenib | Multi-kinase inhibitor | BRAF, VEGFR, PDGFR, KIT | 2007 | First line | NCT00492752 | OS: 10.7 VS 7.9 months (placebo) | (229, 230) |
| Regorafenib | Multi-kinase inhibitor | VEGFR, PDGFR, FGFR1, KIT, RET, BRAF | 2017 | Second line | NCT01774344 | OS: 10.6 VS 7.8 months (placebo) | (7) |
| Nivolumab | ICI | PD-1 | 2017 | Second line | CheckMate-040 CheckMate-459 |
ORR: 20% OS: 16.4 VS 14.7 months (sorafenib) |
(231, 232) |
| Lenvatinib | Multi-kinase inhibitor | VEGFR, FGFR, PDGFR, RET, KIT | 2018 | First line | NCT01761266 | OS: 13.6 VS 12.3 months (sorafenib) | (8) |
| Pembrolizumab | ICI | PD-1 | 2018 | Second line | Keynote-224 Keynote-240 |
ORR: 17% OS: 13.9 VS 10.6 months (placebo) |
(233, 234) |
| Cabozantinib | Multi-kinase inhibitor | VEGFR, MET, RET, KIT, AXL | 2019 | Second line | NCT01908426 | OS: 10.2 VS 8 months (sorafenib) | (6) |
| Ramucircumab | Monoclonal antibody | VEGFR | 2019 | Second line | NCT02435433 | OS: 8.5 VS 7.3 months (placebo) | (9) |
| Nivolumab + Ipilimumab | ICI plus ICI | PD-1 + CTL1-4 | 2020 | Second line | CheckMate-040 | ORR: 33% | (235) |
| Atezolizumab + Bevacizumab | ICI plus anti-VEGF | PD-L1 + VEGF | 2020 | First line | IMbravel | OS: 19.2 VS 13.4 months (sorafenib) | (10) |
ICI, immune checkpoint inhibitor; OS, overall survival; ORR, overall response rate; VEGFR, vascular endothelial growth receptor; PDGFR, platelet-derived growth factor receptor; FGFR, fibroblast growth factor; PD-1, programmed cell death-1; PD-L1, programmed cell death ligand 1; CTLA-4, cytotoxic T lymphocyte-associated antigen-4.