Australian biotechnology company CSL (ASX:CSL; USOTC:CSLLY) announced on Monday that the US Food and Drug Administration (FDA) has approved ANDEMBRY (garadacimab-gxii), the only treatment targeting factor XIIa for prophylactic use to prevent attacks of hereditary angioedema (HAE) in adult and paediatric patients aged 12 years and older.

By targeting factor XIIa, a plasma protein that plays a key role in attacks of swelling in people with HAE, the company says that ANDEMBRY inhibits the top of the HAE cascade to prevent HAE attacks. ANDEMBRY, the only treatment to offer once-monthly dosing from the start for all patients, is a subcutaneous self-injection delivered in 15 seconds or less via an autoinjector with a citrate-free formula.

The approval is supported by data from the placebo-controlled Phase 3 VANGUARD trial evaluating the efficacy and safety of ANDEMBRY. The pivotal study (published in The Lancet, April 2023) demonstrated that treatment with ANDEMBRY: led to 62% of ANDEMBRY-treated patients remaining attack-free throughout the treatment period; reduced HAE attacks by a median of more than 99% and a least squares mean of 89.2%, compared to placebo; achieved more than 99% median reduction and an 88% reduction in HAE attacks requiring on-demand therapy, compared to placebo; and attained more than 99% median reduction and a 90% mean reduction in moderate or severe attacks, compared to placebo. The most common adverse reactions in the pivotal trial (incidence equal to 7%) were nasopharyngitis and abdominal pain.

According to CSL, this regulatory approval is another crucial step in building toward the global availability of ANDEMBRY, which was recently approved in Australia, the UK, the European Union, Japan, Switzerland and the United Arab Emirates.

CSL Behring will immediately launch ANDEMBRY commercially, with availability before the end of June.