CSL receives positive opinion for garadacimab in hereditary angioedema

New treatment could revolutionise care for HAE patients

Global biotechnology leader CSL has announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion on garadacimab, recommending its marketing authorisation as a once-monthly prophylactic treatment for hereditary angioedema (HAE).

If approved, garadacimab will be the first treatment to inhibit factor XIIa, preventing HAE attacks in patients aged 12 years and older.

“CSL has a longstanding and relentless patient-focused approach to developing transformational medicines in areas of unmet need,” said Emmanuelle Lecomte-Brisset, Senior Vice President and Head of Global Regulatory Affairs at CSL. “This CHMP decision brings us closer to offering an innovative treatment to patients living with HAE, which is a debilitating and potentially life-threatening condition.”

HAE attacks are often painful and can affect multiple body areas, including the abdomen, larynx, face, and extremities.

Current therapies work at various steps in the cascade, but garadacimab’s mechanism of action targets the cascade at its start, potentially offering more effective prevention.

The CHMP’s positive opinion is based on data from the pivotal placebo-controlled Phase 3 VANGUARD trial and its open-label extension study.

The pivotal study met its primary endpoint, showing that garadacimab led to 62% of patients achieving attack-free status, reduced the median number of HAE attacks to zero, and decreased the mean number of attacks per month by 86.5% compared to placebo.

Interim analysis of the ongoing extension study showed sustained safety and efficacy over 13.8 months of median exposure.

CSL anticipates the final European Commission decision in Q1 2025. If granted, the marketing authorization for garadacimab would be valid across all EU member states, providing a new, once-monthly treatment option for HAE patients.

“We look forward to making this therapy available to patients in Europe,” added Lecomte-Brisset.