A new hope for hereditary angioedema sufferers
The Committee for Medicinal Products for Human Use (CHMP) of the EMA has issued a positive opinion on garadacimab, an innovative therapy for the prevention of hereditary angioedema (HAE) attacks. Developed by CSL, this monoclonal antibody targets Factor XIIa, a key protein in the cascade that triggers HAE attacks.
The European Commission’s final decision is expected in the first quarter of 2025. Once approved, garadacimab will be available across the EU as a monthly prophylactic treatment for adults and adolescents aged 12 and older.
How garadacimab works
Unlike current therapies targeting downstream mediators, garadacimab prevents the activation of Factor XIIa at the very start of the inflammatory cascade. Results from the phase III VANGUARD trial showed that 62% of patients experienced no attacks, with an 86.5% reduction in attacks compared to placebo.
Data from an ongoing extension study also confirmed long-term safety and efficacy, with attack reduction maintained over a median exposure of 13 months.
CSL’s commitment to HAE
For over 40 years, CSL has supported the HAE community by introducing groundbreaking therapies, such as plasma-derived C1 inhibitors, transitioning from intravenous to subcutaneous formulations. With garadacimab, CSL aims to offer a therapy that could free patients from the symptoms of hereditary angioedema.
“We strive to improve the lives of people with HAE by addressing their needs with innovative solutions,” said Oliver Schmitt, Managing Director of CSL Behring Italy.
What is hereditary angioedema?
Hereditary angioedema is a rare genetic condition affecting approximately 1 in 10,000-50,000 people. It is caused by a deficiency or dysfunction of C1 inhibitor, a protein that regulates inflammation. This leads to fluid buildup in tissues, causing painful swelling in areas such as the face, throat, abdomen, and limbs. If untreated, HAE attacks can be life-threatening, especially when airways are involved.
A brighter outlook
The approval of garadacimab marks a significant milestone in managing hereditary angioedema, offering a safe and effective therapy that could transform the lives of countless patients.
