Table 1.
Drugs Marketed for the Treatment of HAE
| Drug | Trademark | Mechanism | Half-life | Administration route | indications | Dose-adults | Dose-children | Regulatory status |
|---|---|---|---|---|---|---|---|---|
|
Tranexamic acid |
Amchafibrin, Cyklokapron, Lysteda |
Antiplasmin & antiplasminogen activity |
2-8 h | Oral, IV | LTP | 1000-3000 mg/d | 20-40 mg/kg/d |
Europe: approved FDA: NO |
| Lanadelumab | Takhzyro | Kallikrein inhibition | 2 weeks | SC | LTP | 300 mg | Same as adults |
EMA: approved FDA: approved |
| Danazol |
Danatrol, Danocrine |
Increase in hepatic C1-INH synthesis | 7-12 h | Oral |
LTP STP |
200-400 mg/d 400-600 mg/d |
2.5-40 mg/kg/d 5-20 mg/kg/d |
Europe: approved FDA: approved |
| PdC1INH | Cinryze | C1-INH replacement | 56-62 h | IV |
STP On-demand treatment |
1000IU Repeated 1000IU |
500-1000IU Repeated 500-1000IU |
EMA: approved |
| rhCIINH | Ruconest | C1-INH replacement | 3 h | IV |
On-demand treatment |
≤ 84 kg: 50U/kg ＞84 kg: 4200U |
Same as adults |
EMA: approved FDA: approved |
| Icatibant acetate | Firazyr | B2R blockage | 1-2 h | SC |
On-demand treatment |
30 mg | Adjusted by age up to 30 mg |
EMA: approved FDA: approved |
| Ecallantide | Kalbitor | Kallikrein inhibition | 1.5-2.5 h | SC |
On-demand treatment |
3 × 10 mg separated in 3 days | Same as adults |
EMA: NO FDA: approved |
Abbreviations IV-intravenous; SC-subcutaneous; LTP-long term prophylaxis; STP-short term prophylaxis; FDA-United States Food and Drug Administration; EMA- European Medicines Agency.