Last reviewed · How we verify
C1 Inhibitor Human
C1 Inhibitor Human is a plasma-derived protein replacement therapy that restores deficient or dysfunctional C1 esterase inhibitor to prevent excessive bradykinin generation and control hereditary angioedema attacks.
C1 Inhibitor Human is a plasma-derived protein replacement therapy that restores deficient or dysfunctional C1 esterase inhibitor to prevent excessive bradykinin generation and control hereditary angioedema attacks. Used for Hereditary angioedema (HAE) Types I and II — acute attack treatment and prophylaxis.
At a glance
| Generic name | C1 Inhibitor Human |
|---|---|
| Sponsor | GCS Ramsay Santé pour l'Enseignement et la Recherche |
| Drug class | Plasma-derived protease inhibitor |
| Target | C1 esterase inhibitor (C1-INH) |
| Modality | Small molecule |
| Therapeutic area | Immunology / Rare Genetic Disorders |
| Phase | FDA-approved |
Mechanism of action
C1 esterase inhibitor (C1-INH) is a serine protease inhibitor that regulates the contact system and complement cascade. In hereditary angioedema (HAE), deficiency or dysfunction of C1-INH leads to uncontrolled activation of Factor XII and excessive bradykinin production, causing recurrent angioedema attacks. Replacement with human C1-INH protein restores normal regulation of these pathways and prevents acute swelling episodes.
Approved indications
- Hereditary angioedema (HAE) Types I and II — acute attack treatment and prophylaxis
Common side effects
- Thrombosis / thromboembolic events
- Hypersensitivity reactions
- Headache
- Nausea
- Injection site reactions
Key clinical trials
- Targeted Therapy Directed by Genetic Testing in Treating Patients With Advanced Refractory Solid Tumors, Lymphomas, or Multiple Myeloma (The MATCH Screening Trial) (PHASE2)
- Complement and Graft-versus-host Disease (NA)
- Improving Deceased-Donor Kidney Transplant Outcomes Via a Single Intragraft Injection of C1 Esterase Inhibitor (IMPROVE TRIAL) (PHASE2)
- Study of Oral Deucrictibant XR Tablet for Prophylaxis and Deucrictibant IR Capsule for On-Demand Treatment of Angioedema Attacks in Adults With Acquired Angioedema Due to C1 Inhibitor Deficiency (PHASE3)
- A Study to Learn About the C1-Inhibitor Function as Diagnosis for Hereditary Angioedema (NA)
- Study of INBRX-106 and INBRX-106 in Combination With Pembrolizumab (Keytruda®) in Subjects With Locally Advanced or Metastatic Solid Tumors (Hexavalent OX40 Agonist) (PHASE1, PHASE2)
- Dose-ranging Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema (PHASE2)
- A Trial To Find Out If Vidutolimod Together With Cemiplimab Is Safe And If It Works In Adult Participants With Advanced Cancer Or Metastatic Cancer (PHASE2)
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 |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- C1 Inhibitor Human CI brief — competitive landscape report
- C1 Inhibitor Human updates RSS · CI watch RSS
- GCS Ramsay Santé pour l'Enseignement et la Recherche portfolio CI