Last Update: July 31, 2023
| Company | Candidate | MOA | Indication | Administration | Initial Approvals |
|---|---|---|---|---|---|
| BioCryst Pharmaceuticals | Orladeyo (berotralstat) | Oral plasma kallikrein inhibitor | Oral HAE preventative treatment age 12+ | Oral once daily | Dec 2020 (US), Apr 2021 (EU) |
| Takeda Pharmaceuticals | Tahkzyro (lanadelumab) | Anti-Kallikrein mAb | Prophylaxis for HAE age 2+ | SC q2-4 weeks | Aug 2018 (US) Nov 2018 (EU) |
| Takeda Pharmaceuticals | Cinryze (C1-INH) | Plasma-derived C1-INH | Prophylaxis for HAE age 6+ | IV q3-4 days | Oct 2008 (US), Jun 2011 (EU) |
| CSL | Haegarda (C1-INH) | Plasma-derived C1-INH | Prophylaxis for HAE age 6+ | SC q3-4 days | Jun 2017 (US) |
| CSL | Berinert (C1-INH) | Plasma-derived C1-INH | Acute attack of HAE - children, adults | IV-self | Dec 2008 (EU), Oct 2009 (US) |
| Takeda Pharmaceuticals | Kalbitor (ecallantide) | Plasma kallikrein inhibitor | Acute attack of HAE >=12 yrs age | SC | Dec 2009 (US) |
| Takeda Pharmaceuticals | Firazyr (icatibant) | Bradykinin B2 receptor antagonist | Acute attack of HAE >=18 yrs age (EU >=2 yrs) | SC-self | Jul 2008 (EU), Aug 2011 (US) |
| Santarus/Salix Pharmaceuticals | Ruconest (conestat alfa) | Recombinant C1-INH | Acute attack of HAE adolescents, adults | IV-self | Oct 2010 (EU), Jul 2014 (US) |
| Company | Candidate | MOA | Indication | Administration | Phase |
|---|---|---|---|---|---|
| Ionis Pharmaceuticals, Inc. | Donidalorsen (IONIS-PKK-LRx) | Ligand-conjugated anti-sense to target prekallikrein | HAE prophylaxis | SC Q4w | III |
| CSL | Garadacimab (CSL312) | Anti-factor XII mAb | HAE prophylaxis | SC Q4w | III |
| KalVista Pharmaceuticals | KVD900 (Sebetralstat) | Plasma kallikrein inhibitor | On-demand treatment of HAE attacks | Oral | III |
| Pharvaris | PHVS416 | Oral deucrictibant (B2-receptor antagonist) | On-demand HAE | Oral | III |
| Pharvaris | PHVS416 | Oral deucrictibant (B2-receptor antagonist) | HAE prophylaxis | Oral | II |
| Astria | STAR-0215 | Anti-Kallikrein mAb | HAE prophylaxis | SC | Ib/II |
| Intellia Therapeutics | NTLA-2002 | LNP-CRISPR to inactivate Kallikrein B1 gene (KLKB1) | HAE prophylaxis | Single-dose IV | I/II |
| BioMarin | BMN 331 | AAV5 gene therapy for wt hC1-INH expression | HAE prophylaxis | Single-dose IV | I/II |
| Pharvaris | PHVS719 | Oral deucrictibant XR release (B2-receptor antagonist) | HAE prophylaxis | Oral | I |
| ADARx | ADX-324 | siRNA to reduce PKK production | HAE prophylaxis | SC | I |
| Attune Pharmaceuticals | ATN-249 | Kallikrein inhibitor | HAE prophylaxis | Oral | I |
| KalVista Pharmaceuticals | - | Oral Factor XIIa | HAE prophylaxis | Oral | IND:2023 |
| Pharming/Orchard Therapeutics | OTL-105 | ex-vivo HSC gene therapy | HAE prophylaxis | IV | Preclin |
| Verseon | VE-4666 | Plasma kallikrein inhibitor | HAE prophylaxis | Oral | Preclin |
| Verseon | VE-4062 | Plasma kallikrein inhibitor | HAE prophylaxis | Oral | Preclin |
| Bridge Medicines | - | Factor XIIa inhibitors | HAE | - | Preclin |
| Octapharma Pharmazeutika Produktionsges.m.b.H | OCTA-C1-INH | human plasma derived C1-inhibitor | Pediatric on-demand treatment | IV | Preclin |