After gaining approvals in Japan, Europe and the U.K. for its hereditary angioedema (HAE) preventative treatment Andembry, CSL has scored a nod for the monoclonal antibody in the United States.
With the FDA signing off on Andembry, CSL is ready to take on Takeda’s blockbuster Takhzyro and BioCryst Pharmaceuticals’ fast-rising Orladeyo in a competitive market to treat patients with the rare genetic condition.
HAE, which strikes 1 in 50,000, causes sudden, unpredictable episodes of severe swelling in the limbs, face, abdomen and larynx. The disorder can be fatal when it strikes the airways and inhibits breathing.
Andembry, which is self-administered monthly with a prefilled pen, is the first prophylactic treatment that inhibits factor XIIa, a plasma protein that plays a key role in triggering the swelling episodes. The FDA has approved it to prevent HAE attacks—rather than on-demand treatment for acute attacks—in patients age 12 and older.
“We’ve made significant progress in treating hereditary angioedema, yet many patients still experience painful and sometimes life-threatening HAE attacks and require frequent injections to manage them,” Tim Craig, M.D., a professor of medicine at Penn State University, said in a release. “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.”
In a phase 3 trial, Andembry significantly reduced investigator-confirmed HAE attacks per month versus placebo, according to results published in The Lancet in April of 2023. Of the 39 patients who received Andembry, 62% remained attack-free over the six-month study period.
The study suggested that Andembry has a convenience and efficacy edge over bi-weekly Takhzyro with the caveat of cross-trial comparisons. Last year, Takhzyro generated sales of $1.2 billion, while BioCryst’s oral HAE drug Orladeyo accounted for sales of $438 million.
Both therapies have taken market share from CSL’s HAE treatments Berinert, which was approved in 2009, and Haegarda, which earned its FDA nod in 2017. CSL does not break down sales figures for individual products.
CSL said that it will make Andembry available by the end of this month.
“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,” Bill Mezzanotte, M.D., CSL’s R&D chief, said in a release.