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CSL's Revolutionary HAE Treatment: ANDEMBRY® Approved by FDA

On June 16, 2025, CSL, a prominent global biotechnology firm, announced that the U.S. Food and Drug Administration (FDA) has approved ANDEMBRY® (garadacimab-gxii), marking a groundbreaking advancement in the treatment of hereditary angioedema (HAE). This innovative therapy is specifically designed to target factor XIIa, a crucial protein involved in the swelling attacks associated with HAE. ANDEMBRY® is remarkable not only for its efficacy but also for its unique once-monthly dosing regimen, making it the first of its kind for both adults and children aged 12 years and older.

Understanding Hereditary Angioedema (HAE)

HAE is a rare but life-threatening genetic condition that affects approximately 1 in 50,000 people, causing recurrent and often unpredictable episodes of angioedema. These attacks can lead to significant swelling in different body parts, including the face, abdomen, and throat, posing risks of asphyxiation if left untreated.

Due to the unpredictable nature of HAE, patients have faced challenges in managing their symptoms, often requiring multiple interventions and treatments. ANDEMBRY® aims to change that by offering a more manageable and effective option.

ANDEMBRY®: How It Works

ANDEMBRY® functions by inhibiting factor XIIa, a plasma protein that initiates the cascade of effects leading to HAE attacks. This mechanism provides a novel approach to treatment, as most existing therapies target mechanisms further down the HAE pathway. The subcutaneous injection can be self-administered in under 15 seconds using an autoinjector and contains a citrate-free formulation, catering to the needs of patients who prefer a simplified treatment process.

Clinical trials have demonstrated impressive results, with the pivotal Phase 3 VANGUARD trial showing:

• - 62% of patients remained attack-free throughout the trial.
• - 99% median reduction in attack frequency compared to placebo, demonstrating unparalleled effects in managing HAE episodes.
• - Significant declines in moderate or severe attacks, further solidifying ANDEMBRY®'s efficacy.

A Long-awaited Leap Forward

Dr. Bill Mezzanotte, CSL’s Executive Vice President, expressed that ANDEMBRY® embodies the company’s unwavering commitment to enhance the quality of life for HAE patients. “This groundbreaking treatment offers substantial long-term control over HAE and provides a convenient administration method,” he stated.

Furthermore, Anthony J. Castaldo, CEO of the US HAE Association, welcomed this new option, noting, “It is vital for patients to have therapies that alleviate their burdens while allowing them to lead fulfilling lives.”

Global Recognition

The approval of ANDEMBRY® is not only celebrated in the United States but also marks an essential milestone in its global rollout. Earlier approvals in countries such as Australia, the UK, and various European nations indicate that CSL is poised for a wider distribution, ultimately benefiting a larger patient population.

Healthcare professionals are encouraged to explore ANDEMBRY ConnectSM, a support program designed to assist patients in accessing the therapy and understanding its various benefits. For more information, patients or healthcare providers can visit www.ANDEMBRY.com or call 844-423-4273.

Conclusion

As a groundbreaking advancement in the fight against hereditary angioedema, ANDEMBRY® signifies a pivotal moment for both patients and healthcare providers. It serves not only as a potential lifesaver for many but also as a model for future innovations in the treatment of rare genetic disorders. As CSL embarks on the journey to make ANDEMBRY® widely available, the hope for improved quality of life and better disease management shines brighter than ever.