Australia-based global biotechnology company CSL (ASX:CSL; USOTC:CSLLY) announced on Monday 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 paediatric patients aged 12 years and older.

In this multicentre, open-label Phase 3b study, ANDEMBRY demonstrated a favourable 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.

CSL say that it plans to begin submitting filings to health authorities in the first half of the company's fiscal year to support an expanded paediatric 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.