Australian biotech CSL is heading to regulators after the company’s marketed hereditary angioedema (HAE) therapy, Andembry (garadacimab), demonstrated its promise in treating paediatric patients with the rare swelling disorder – supporting the drug’s potential expansion into a new patient population.
During an open-label Phase IIIb study (NCT05819775), which enrolled HAE patients aged between two and 11, Andembry – a subcutaneous factor-XIIa-targeting therapy, triggered treatment responses “across the study population,” the company claimed.
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Though CSL is yet to disclose details on Andembry’s efficacy in this study, the biotech has noted that the majority of treated patients remained free of swelling attacks during the 12-month trial.
Patients treated with Andembry also tolerated the drug well, with the therapy’s safety profile remaining consistent with previous studies.
According to Dr Bill Mezzanotte, CSL’s EVP and head of R&D, these topline results support the company’s plans to seek a label expansion into the paediatric population for Andembry in children aged two to 11 years of age. The company plans to file for the therapy’s expanded approval in HAE in the first half of its fiscal year.
“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,” Mezzanotte said.
HAE is a rare genetic condition where patients have a deficient or dysfunctional version of a specific inflammation-controlling protein in the blood, which can often lead to a buildup of fluid and swelling in different areas of the body. Treating clinicians employ both preventative therapies like Andembry as well as acute treatments like C1 esterase inhibitors to manage the disease, which impacts between one in 10,000 and one in 50,000 patients globally.
HAE market sees new players
While HAE does continue to place a burden on patients, there are now several therapeutic options available to treat the condition, owing to recent approvals in the space.
If Andembry were to reach the paediatric HAE market, it would further extend CSL’s legacy in this indication, which is currently defined by Andembry and its two C1 esterase inhibitors, Berinert and Haegarda.
On the paediatric HAE prevention market, Andembry’s toughest competition would currently be Takeda’s blockbuster subcutaneous therapy, Takhzyro (lanadelumab), which is the top seller in this indication, according to GlobalData’s Pharmaceutical Intelligence Center.
However, the drug would also face competition from BioCryst’s Orladeyo (berotalstat), which became the first oral therapy to secure approval in the paediatric population for the prevention of HAE attacks back in December 2025. While GlobalData analysts forecast that Orladeyo will near the blockbuster mark, they predict that Takhzyro will remain the best-seller in HAE, with analysts forecasting sales of just under $1.5bn in 2032.
In a recent comment piece, GlobalData analysts noted that emerging technologies such as CRISPR and siRNA platforms could offer differential potential across the increasingly competitive HAE landscape.
