CSL receives positive CHMP opinion for garadacimab in HAE

CSL (CSLLY) announced the European Medicines Agency’s Committee for Medicinal Products for Human Use adopted a positive opinion, recommending granting a marketing authorization for garadacimab as a once-monthly prophylactic treatment for hereditary angioedema in adult and adolescent patients aged 12 years and older. Garadacimab is a monoclonal antibody that targets activated factor XII, a plasma protein that plays a key role in attacks of swelling in people with HAE, thereby inhibiting the HAE cascade at the top to prevent HAE attacks. The final European Commission decision is expected in Q1 2025. The CHMP positive opinion is based on data from the pivotal placebo-controlled Phase 3 VANGUARD trial and its open-label extension study, both evaluating the efficacy and safety of garadacimab. The pivotal study met its primary endpoint and showed that garadacimab led to 62 percent of patients achieving attack-free status throughout the treatment period, reduced the median number of HAE attacks to zero and reduced the mean number of HAE attacks per month by 86.5 percent compared to placebo. Interim analysis of the ongoing open-label extension study showed that garadacimab has a favorable long-term safety profile and provides sustained HAE attack reduction. The full results from VANGUARD were published in The Lancet and the primary results of the ongoing open-label extension study were published in Allergy.