FDA Approves Monthly Treatment Andembry for Hereditary Angioedema

The U.S. Food and Drug Administration (FDA) has granted approval to Andembry (garadacimab-gxii), a novel medication designed for the prevention of hereditary angioedema (HAE) attacks. Notably, this medication is the first in its class that specifically targets factor XIIa, a key plasma protein involved in swelling episodes associated with HAE. Administration is convenient, with patients able to self-inject via an autoinjector in just 15 seconds, making it suitable for both adults and children aged 12 and older.

Hereditary angioedema is characterized by sudden, severe swelling typically affecting the face, throat, and extremities, often accompanied by symptoms such as swelling around eyes and cheeks, or lips, and mild pain. According to the Mayo Clinic, it manifests as deeper swelling of the skin, sometimes with hives, and can develop within minutes to hours.

The approval was based on results from a phase 3 clinical trial, wherein 62% of patients treated with Andembry experienced no attacks over six months. The medication demonstrated a median reduction of over 99% in HAE attacks compared to placebo, as well as significant decreases in attacks requiring on-demand treatment and moderate to severe attacks (both around 90-99%). Common side effects included nasal and upper throat inflammation, along with abdominal pain.

Bill Mezzanotte, M.D., CSL's head of research and development, highlighted that Andembry represents a groundbreaking advancement, offering individuals with HAE long-term disease management via a simple and effective method. CSL was the company granted approval for this innovative therapy.

For more details, visit: https://newsroom.csl.com/2025-06-16-U-S-Food-and-Drug-Administration-Approves-CSLs-ANDEMBRY-R-garadacimab-gxii-,-the-Only-Prophylactic-Hereditary-Angioedema-HAE-Treatment-Targeting-Factor-XIIa-with-Once-Monthly-Dosing-for-All-Patients-From-the-Start