Overview of FDA Approval

The U.S. Food and Drug Administration has approved CSL’s ANDEMBRY (garadacimab-gxii), marking the first prophylactic therapy to specifically target factor XIIa for hereditary angioedema (HAE). This approval covers patients aged 12 and older and represents a significant advancement in HAE prophylaxis options. ANDEMBRY is administered once monthly through a subcutaneous autoinjector, providing convenient and durable protection against potentially life-threatening HAE attacks.

Clinical Evidence Supporting Approval

The approval is based on data from the Phase 3 VANGUARD trial, which demonstrated a median reduction of ≥99% in HAE attack frequency compared to placebo. Notably, 62% of patients receiving ANDEMBRY remained attack-free during the treatment period. The drug’s safety profile was favorable, with most adverse events being mild to moderate injection site reactions.

Mechanism of Action

ANDEMBRY is a fully human recombinant monoclonal antibody that selectively inhibits activated factor XII (factor XIIa), a critical initiator of the kallikrein-kinin pathway involved in HAE pathogenesis. By blocking factor XIIa, ANDEMBRY prevents excessive bradykinin production, reducing vascular permeability and HAE attack occurrence.

Clinical and Market Implications

This approval expands the therapeutic arsenal for HAE patients, especially those seeking less frequent dosing schedules and improved quality of life. CSL anticipates that ANDEMBRY’s unique mechanism and ease of administration will foster broad adoption in the prophylactic treatment landscape.

Related Regulatory and Industry Developments

June 2025 has seen a spate of notable approvals in rare and chronic diseases, highlighting regulatory agencies’ focus on patient-centered innovations. CSL’s milestone adds to the momentum in recombinant antibody therapies addressing unmet needs.