Table 1.
Approved treatments for hereditary angioedema.
| Treatment | Route of administration | FDA-approved indication and dose | EMA-approved indication(s) and dose | MHLW-approved indication(s) and dose |
|---|---|---|---|---|
| C1-INH replacement products | ||||
| pdC1-INH (Berinert) | IV | Acute treatment in pediatric patients and adults (20 IU/kg) | Acute treatment (20 IU/kg) and short-term prophylaxis (pediatric patients: 15–30 IU/kg; adults: 1,000 IU) | Acute treatment and short-term prophylaxis (<50 kg: 500 U; >50 kg, 1,000–1,500 U) |
| pdC1-INH (Cinryze) | IV | Long-term prophylaxis in patients aged ≥6 y (children 6–11 y: 500 U q3–4 d; adolescents ≥12 y and adults: 1,000 U q3–4 d) | Acute treatment (2–11 y [10–25 kg]: 500 IU; 2–11 y [>25 kg: 1,000 IU]; ≥12 y: 1,000 IU) and short-term prophylaxis in patients aged ≥2 y (2–11 y [10–25 kg]: 500 IU <24 h; 2–11 y [>25 kg]: 1,000 IU <24 h; ≥12 y: 1,000 IU <24 h); long-term prophylaxis in patients aged ≥6 y (6–11 y: 500 IU q3–4 d; ≥12 y: 1,000 IU q3–4 d) | Not approved |
| rhC1-INH/Conestat alfa (Ruconest) | IV | Acute treatment in adolescents and adults (<84 kg: 50 U/kg; ≥84 kg: 4,200 U) | Acute treatment in patients aged ≥2 y (<84 kg: 50 U/kg; ≥84 kg: 4,200 U) | Not approved |
| pdC1-INH (Haegarda [US]/Berinert [EU]) | SC | Long-term prophylaxis in patients aged ≥6 y (60 IU/kg q3–4 d) | Long-term prophylaxis in adolescents and adults (60 IU/kg) | Not approved |
| Bradykinin B2 receptor antagonist | ||||
| Icatibant (Firazyr) | SC | Acute treatment in patients aged ≥18 y (30 mg) | Acute treatment in patients aged ≥2 y (patients 2–17 y: 12–25 kg, 10 mg; 26–40 kg, 15 mg; 41–50 kg, 20 mg; 51–65 kg, 25 mg; >65 kg, 30 mg; adults: 30 mg) | Acute treatment in adults (30 mg) |
| Plasma kallikrein inhibitors | ||||
| Ecallantide (Kalbitor) (33) | SC | Acute treatment in patients aged ≥12 y (30 mg) | Not approved | Not approved |
| Lanadelumab (Takhzyro) (34, 35) | SC | Long-term prophylaxis in patients aged ≥12 y (300 mg q2–4 wk) | Long-term prophylaxis in patients aged ≥12 y (300 mg q2–4 wk) | Long-term prophylaxis in patients aged ≥12 y (300 mg q2–4 wk) |
| Berotralstat (Orladeyo) (36, 37) | Oral | Long-term prophylaxis in patients aged ≥12 y (150 mg once daily) | Long-term prophylaxis in patients aged ≥12 y (150 mg once daily) | Long-term prophylaxis in patients aged ≥12 y (150 mg once daily) |
EMA, European Medicines Agency; FDA, US Food and Drug Administration; IV, intravenous; MHLW, Ministry of Health, Labour and Welfare (Japan); pdC1-INH, plasma-derived C1 esterase inhibitor; SC, subcutaneous.