MedPath

Human C1-esterase inhibitor

Generic Name
Human C1-esterase inhibitor
Brand Names
Berinert, Cinryze, Haegarda
Drug Type
Biotech
Chemical Formula
-
CAS Number
-
Unique Ingredient Identifier
6KIC4BB60G
Background

C1 Esterase Inhibitor (Human) is composed of purified endogenous complement component-1 esterase inhibitor (hC1INH) isolated from human plasma. The primary function of endogenous C1INH is to regulate the activation of the complement and contact system pathways.

This drug is indicated for prophylaxis and treatment of Hereditary Angioedema (HAE), a human genetic disorder caused by a shortage of C1 inhibitor activity that results in an overreaction of the immune system. The disease is characterized by acute attacks of painful, and in some cases, fatal swelling of several soft tissues or edema, which may last up to five days when untreated.

Indication

Intravenous and subcutaneous formulations of the human C1-esterase inhibitor are indicated for routine prophylaxis against acute attacks of hereditary angioedema in patients six years of age and older. It is also used to treat these in adult and adolescent patients with hereditary angioedema.

Associated Conditions
Acute attacks of hereditary angioedema, Acute attack of hereditary angioedema of the Abdomen, Acute attack of hereditary angioedema of the Larynx, Acute attack of hereditary angioedema of the face
Associated Therapies
-
nature.com
·

Genetically engineered pig heart transplantation in non-human primates and humans shows promise for end-stage heart failure

Clinical cardiac xenotransplantation using genetically engineered pig hearts shows promise for end-stage heart failure patients. Recent transplants of 10-GE pig hearts into humans and non-human primates demonstrated survival up to 225 days, indicating potential to alleviate donor organ shortages. Gene edits aimed at preventing rejection and controlling inflammation were key to prolonged xenograft survival.
pharmaphorum.com
·

Phase 3 trial win sets up rare disease drug filing for CSL

CSL's garadacimab, a once-monthly therapy for hereditary angioedema (HAE), shows efficacy comparable to Takeda's Takhzyro, with 86.5% reduction in monthly attacks. Garadacimab's less frequent dosing could challenge Takhzyro's market dominance, alongside BioCryst's Orladeyo. CSL also markets Berinert and Haegarda for HAE, facing growth challenges from new therapies.
angioedemanews.com
·

Top 10 news stories of 2024 on rare disease angioedema

In 2024, Angioedema News highlighted top advancements in angioedema treatment, including deals for donidalorsen, NTLA-2002's 95% attack reduction, Takeda's partnerships, Takhzyro's efficacy in adolescents, deucrictibant's Phase 3 trials, FDA's lift on deucrictibant hold, Ionis' FDA application for donidalorsen, garadacimab's safety, Haegarda's real-world effectiveness, and Orladeyo's long-term benefits in Japan.
healthline.com
·

New Treatments for Hereditary Angioedema

Hereditary angioedema (HAE) treatment options have expanded with eight FDA-approved medications and more in development. HAE, a rare genetic disorder, involves C1 esterase inhibitor gene mutations causing severe swelling. While no cure exists, therapies like kallikrein inhibitors (Lanadelumab-flyo, Berotralstat, Ecallantide) and C1 esterase inhibitors (Cinryze, Haegarda, Ruconest, Berinert) aim to prevent and manage attacks.
medicalresearch.com
·

HAE: The Latest in Clinical Research

Clinical research advancements in Hereditary Angioedema (HAE) are improving treatment options, emphasizing early diagnosis, patient education, personalized medicine, and overcoming development challenges. Staying updated through medical journals, conferences, and professional networks is crucial for healthcare professionals.
© Copyright 2025. All Rights Reserved by MedPath