Andembry treatment approved across Europe

Significant progress for hereditary angioedema condition

Global biotechnology leader CSL has announced that the European Commission (EC) has approved ANDEMBRY (garadacimab), the first once-monthly treatment targeting factor XIIa to prevent hereditary angioedema (HAE) attacks in patients aged 12 and older.

ANDEMBRY inhibits plasma protein factor XIIa, which starts the cascade of events leading to angioedema at various body sites.

CSL’s Bill Mezzanotte, Executive Vice President, Head of R&D, commented, “ANDEMBRY is a significant advancement in the management of hereditary angioedema, offering long-term control over their disease with a patient-centric, convenient administration method.

ANDEMBRY underscores our more than 40-year legacy in HAE research and treatment optimization and our decades-long journey to bring this innovation to patients.”

HAE is a rare, chronic and life-threatening genetic disorder characterised by recurrent attacks of angioedema, often painful and can spread to multiple body sites, including the abdomen, larynx, face and extremities. It occurs in about 1 in 50,000 people of any ethnic group.

Markus Magerl, Head of Clinical Trials at Charité University Hospital Berlin, said, “The physical and emotional toll of HAE is substantial and the true prevalence could be higher than recorded due to misdiagnoses. The unpredictable nature of HAE is daunting as patients never know when an attack might occur. Current HAE preventive therapies work at downstream steps in the cascade but none prevent the cascade at its start like ANDEMBRY.”

The approval is based on efficacy and safety data from the pivotal international phase 3 VANGUARD trial and its open-label extension study. ANDEMBRY is now under review by regulatory agencies in the United States, Japan, Switzerland and Canada.