Healthcare
CSL Reports Positive Top-Line Phase 3b Results Supporting Planned Expanded Pediatric Filing for ANDEMBRY® (garadacimab-gxii) in Children with Hereditary Angioedema (HAE)
Global biotechnology leader CSL (ASX:CSL; USOTC:CSLLY) today announced positive top-line Phase 3b results evaluating ANDEMBRY® (garadacimab-gxii) in children aged 2 to 11 years with hereditary angioedema (HAE). ANDEMBRY is approved in over 40 countries for the prevention of HAE attacks in adult and pediatric patients aged 12 years and older.
About this update from Csl Limited
KING OF PRUSSIA, Pa., July 27, 2026 /PRNewswire/ -- Global biotechnology leader CSL (ASX:CSL; USOTC:CSLLY) today announced positive top-line Phase 3b results evaluating ANDEMBRY® (garadacimab-gxii) in children aged 2 to 11 years with hereditary angioedema (HAE). ANDEMBRY is approved in over 40 countries for the prevention of HAE attacks in adult and pediatric patients aged 12 years and older. In this multicenter, open-label Phase 3b study evaluating ANDEMBRY in people living with HAE aged 2 to 11 years, ANDEMBRY demonstrated a favorable safety and tolerability profile consistent with previous studies. Treatment response was observed across the study population, with the majority of participants remaining attack-free during the 12-month treatment period. Participants aged 6 to 11 years (n = 16) received 100 mg of ANDEMBRY once monthly, while participants aged 2 to 5 years (n = 6) received 100 mg of ANDEMBRY every two months. ANDEMBRY is a monoclonal antibody that targets factor XIIa, a plasma protein that plays a key role in attacks of swelling in people with HAE. By acting at the top of the HAE cascade, ANDEMBRY prevents attacks and represents a novel long-term prophylactic strategy. "The top-line results support our plans to seek an expanded pediatric indication for ANDEMBRY in children aged 2 to 11 years," said Dr. Bill Mezzanotte, Executive Vice President, Head of R&D, CSL. "We are excited to share the full study findings with the scientific and patient communities at an upcoming medical congress and remain committed to advancing treatment options for people living with hereditary angioedema." CSL plans to begin submitting filings to health authorities in the first half of the company's fiscal year to support an expanded pediatric indication for ANDEMBRY in children aged 2 to 11 years. Full study results will be presented at an upcoming scientific congress and submitted for publication in a peer-reviewed journal. About Hereditary Angioedema (HAE) HAE is a rare and potentially life-threatening genetic condition that occurs in about 1 in 10,000 to 1 in 50,000 people. HAE is caused by deficient or dysfunctional C1 esterase inhibitor (C1INH), a protein in the blood that helps to control inflammation. Inadequate amounts of properly functioning C1INH can lead to the accumulation of fluid in body tissues, causing considerable swelling referred t...