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C1 Esterase Inhibitor (Human)
C1 esterase inhibitor (C1-INH) suppresses activation of the contact system and complement cascade by inhibiting plasma kallikrein and Factor XIIa, thereby reducing bradykinin generation and preventing angioedema attacks.
C1 Esterase Inhibitor (Human) is a marketed drug by Shire for treating Hereditary Angioedema (HAE) in adults and pediatric patients. It prevents HAE attacks and treats existing attacks. The drug has 59 trials and 8 publications. Its commercial significance is notable, but the mechanism is not specified. This drug is a key treatment option for HAE patients. Shire is the company behind this drug. There are no pipeline developments mentioned.
At a glance
| Generic name | C1 Esterase Inhibitor (Human) |
|---|---|
| Also known as | C1 INH-nf |
| Sponsor | Shire |
| Drug class | Serine protease inhibitor; complement regulator |
| Target | C1 esterase inhibitor (C1-INH); Factor XIIa; plasma kallikrein |
| Modality | Biologic |
| Therapeutic area | Immunology; Rare Genetic Disorders |
| Phase | FDA-approved |
Mechanism of action
C1-INH is a serine protease inhibitor that regulates the intrinsic coagulation pathway and complement system. In hereditary angioedema (HAE), deficiency or dysfunction of C1-INH leads to uncontrolled activation of Factor XII and kallikrein, resulting in excessive bradykinin production and vascular permeability. Replacement with human C1-INH restores normal regulation of these cascades, preventing acute angioedema episodes and reducing their frequency and severity.
Approved indications
- Hereditary angioedema (HAE) Types I and II — acute attack treatment
- Hereditary angioedema — prophylaxis of recurrent attacks
Common side effects
- Thrombosis
- Headache
- Nausea
- Injection site reactions
- Fever
Key clinical trials
- Complement and Graft-versus-host Disease (NA)
- Study of IV Human Plasma-derived C1 Esterase Inhibitor Concentrate in Patients With Congenital C1-INH Deficiency for Treatment and Pre-procedure Preventing of Acute Hereditary Angioedema Attacks (PHASE3)
- C1 Inhibitor Registry in the Treatment of Hereditary Angioedema (HAE) Attacks
- Recombinant Human C1 Esterase Inhibitor (Conestat Alfa) in the Prevention of Acute Ischemic Cerebral and Renal Events After Transcatheter Aortic Valve Implantation (PHASE2)
- Complement Inhibition: Attacking the Overshooting Inflammation @Fter Subarachnoid Hemorrhage (CIAO@SAH) (PHASE2)
- Berinert (C1INH) vs Placebo for DGF/IRI (PHASE1, PHASE2)
- Treatment of Hereditary Angioedema Prodrome with Recombinant C1-esterase Inhibitor (Ruconest) (PHASE4)
- Safety and Efficacy of Conestat Alfa for ACE-Induced Angioedema (PHASE3)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |