Garadacimab

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.

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.

EMA's CHMP recommended garadacimab for HAE, ages 12+, as a once-monthly preventive treatment. The European Commission will review the recommendation, with a decision expected in Q1 2025. Garadacimab, designed to block FXII activation, reduced swelling attack rates by over 85% in a Phase 3 trial, with ongoing extension study showing 95% reduction and half of patients free from attacks. Common side effects were injection site reactions.

EMA's CHMP recommended 17 drugs for market authorization in Dec 2024, including 5 orphan drugs and 6 biosimilars. Notable recommendations include Kavigale for COVID-19 prevention in immunocompromised individuals and Kostaive, a mRNA vaccine for COVID-19 prevention. CHMP also extended recommendations for several drugs and withdrew Alofisel from the market.

CSL announced EMA's CHMP recommended garadacimab for HAE, a once-monthly treatment inhibiting factor XIIa. The decision is based on Phase 3 VANGUARD trial data showing 62% attack-free status and 86.5% reduction in attacks. The EC decision is expected in Q1 2025.

EU decision on marketing approval for 13 new products, including BridgeBio’s acoramidis, is imminent.