Imge source: Shutterstock

Highlights

  • ANDEMBRY® is the first and only prophylactic treatment targeting factor XIIa approved in the U.S. for hereditary angioedema (HAE).

  • FDA approval follows previous regulatory clearances in the UK, EU, Australia, Japan, Switzerland, and UAE.

  • CSL plans immediate U.S. launch, with product availability expected before the end of June.

CSL (ASX:CSL; USOTC:CSLLY) has announced that the U.S. Food and Drug Administration (FDA) has approved ANDEMBRY® for immediate launch across the United States. ANDEMBRY® is now officially authorized as a preventive treatment for hereditary angioedema (HAE) in adult and pediatric patients aged 12 years and older.

HAE is a rare and potentially life-threatening genetic disorder that causes recurrent episodes of severe swelling in various parts of the body. It affects approximately 1 in 50,000 individuals across all ethnic groups.

First-of-Its-Kind Treatment Targeting Factor XIIa

ANDEMBRY® is the first and only prophylactic therapy that works by targeting factor XIIa, a key component in the HAE disease cascade. This novel mechanism provides sustained protection against HAE attacks, offering patients a new therapeutic option distinct from existing treatments.

Unlike other HAE therapies, ANDEMBRY® is administered once a month via an autoinjector and is suitable for all eligible patients from the beginning of treatment. According to CSL, this unique approach addresses both patient convenience and adherence.

Clinical Results from Phase 3 VANGUARD Trial

The FDA’s approval was based on findings from the pivotal Phase 3 VANGUARD clinical trial, a placebo-controlled study, along with its open-label extension. The studies demonstrated that ANDEMBRY® reduced the median number of HAE attacks by more than 99% and showed a least squares mean reduction of 89.2% in comparison to placebo.

Global Regulatory Momentum and U.S. Launch Timeline

This U.S. FDA approval follows a series of global regulatory authorizations for ANDEMBRY®, including approvals in Australia, the United Kingdom, the European Union, Japan, Switzerland, and the United Arab Emirates. The company stated that these approvals reflect growing international confidence in the therapy’s safety and efficacy profile.

CSL confirmed that ANDEMBRY® will be launched in the United States immediately. The treatment is expected to be accessible through CSL’s third-party specialty pharmacy network before the end of June.

Market Response

Following the FDA announcement, shares of CSL rose by 0.62% to AUD 240.59 during Tuesday morning trading.