EU approves CSL's once-monthly HAE drug Andembry

CSL has been given approval by the European Commission for Andembry, the first once-monthly preventive therapy for the attacks that characterise rare genetic disorder hereditary angioedema (HAE).

Factor XIIa inhibitor Andembry (garadacimab) has been cleared for use in patients aged 12 and over with HAE, which affects about 1 in 10,000 to 1 in 50,000 people worldwide and leads to unpredictable bouts of debilitating and painful swelling in various parts of the body. If the swelling affects the airways it can lead to asphyxiation and be life-threatening.

Andembry, which is administered subcutaneously using an auto-injector pen, provides less frequent dosing Takeda's big-selling kallikrein inhibitor Takhzyro (lanadelumab), which requires a subcutaneous injection every two weeks, and an alternative to Biocryst's daily oral kallikrein inhibitor Orladeyo (berotralstat) that was cleared in the EU in 2021.

CSL's drug – which is also the first Factor XIIa inhibitor to be approved for HAE – is a successor to its older HAE prevention products Berinert, given intravenously, and Haegarda which is given twice weekly as a subcutaneous injection. Takeda also sells an older IV therapy called Cinryze, but these products, all C1 esterase inhibitors, have seen their sales come under pressure from Takhzyro and Orladeyo.

Takhzyro is already a blockbuster with sales of JPY 168 billion ($1.1 billion) in the nine months ending 31st December 2024, up around 16%, while Biocryst reported Orladeyo sales rose 34% to $437 million in 2024 and it expects to book up to $535 million from the product this year.

Andembry was shown to reduce the average number of monthly attacks by 86.5% compared to placebo in the VANGUARD study reported in 2023, with almost two-thirds of patients (61.5%) taking CSL's drug attack-free over the six-month study period compared to zero in the control group.

The drug has also been approved in the UK and Australia to date and is under regulatory review in the US, China, Japan, and Switzerland, according to CSL.

CSL's head of R&D, Paul Mezzanotte, said that Andembry offers "a patient-centric, convenient administration method" that will help HAE patients achieve "long-term control over their disease."

It will be launched in the EU by the company's CSL Behring unit upon completion of access and reimbursement negotiations.

Henrik Balle Boysen, president of the patient advocacy organisation HAE International (HAEi), said that Andembry is "a welcome addition to the HAE treatment landscape" and "another choice for lessening the burden associated with this lifelong condition and realising the community's shared goal of experiencing life to the fullest."