The US Food and Drug Administration (FDA) has approved CSL’s Andembry (garadacimab-gxii) to prevent attacks of hereditary angioedema (HAE), a rare and potentially life-threatening genetic disorder affecting approximately one in 10,000 to one in 50,000 people worldwide.
HAE is characterised by unpredictable, and often painful, attacks of swelling (angioedema) that can spread to multiple parts of the body, including the abdomen, face, hands and feet, as well as the airways and gastrointestinal tract.
CSL’s Andembry, which has been authorised for use in adult and paediatric patients aged 12 years and older, is a monoclonal antibody designed to target factor XIIa, a plasma protein that plays a crucial role in initiating attacks of swelling in HAE.
The drug is self-administered subcutaneously with an auto-injector and is the only treatment to offer once-monthly dosing from the start for all patients, the company said.
The FDA’s decision was supported by positive results from the phase 3 VANGUARD trial, in which 62% of patients receiving Andembry achieved attack-free status throughout the treatment period. Andembry was shown to reduce HAE attacks by a median of more than 99% and a least squares mean of 89.2% compared to placebo.
Data from an ongoing open-label extension study also demonstrated that the drug has a favourable long-term safety profile and provides sustained reductions in HAE attacks.
“Andembry, the first monoclonal antibody discovered and developed entirely by CSL, offers people living with this life-threatening condition long-term control over their disease along with a convenient administration method,” said Bill Mezzanotte, executive vice president, head of research and development at CSL.
The company’s CSL Behring business unit will launch Andembry immediately, with availability before the end of June.
Welcoming the approval, Tim Craig, professor of medicine, paediatrics and biomedical sciences at Penn State University, said: “We’ve made significant progress in treating HAE, yet many patients still experience painful and sometimes life-threatening HAE attacks and require frequent injections to manage them.
“We now have a new option to manage this condition through a new target, as it allows us for the first time to inhibit the top of the HAE cascade by targeting factor XIIa.”
