FDA Grants Approval for Monthly Andembry to Treat Hereditary Angioedema

The U.S. Food and Drug Administration (FDA) has announced the approval of Andembry (garadacimab-gxii), marking it as the first treatment specifically targeting factor XIIa for the prophylactic management of hereditary angioedema (HAE). This condition is characterized by recurrent episodes of severe swelling, which can be life-threatening.

Hereditary angioedema is described by the Mayo Clinic as a condition that leads to swelling in deeper layers of the skin, similar to hives. This swelling can occur with or without accompanying hives and typically manifests as welts and swelling, particularly around sensitive areas such as the eyes, lips, and cheeks. Symptoms can develop rapidly, within minutes to hours, and may involve mild pain.

Andembry is approved for use in both adult and pediatric patients aged 12 and older. The medication is designed for self-administration, requiring only a once-monthly injection that can be delivered in under 15 seconds via an autoinjector. Notably, this formulation is citrate-free, which may enhance patient comfort during administration.

The FDA's approval was based on findings from a pivotal phase 3 clinical trial. Results indicated that approximately 62% of patients receiving Andembry remained free from HAE attacks throughout the six-month study period. Moreover, Andembry demonstrated a median reduction of over 99% in the frequency of HAE attacks when compared to a placebo. Additional findings revealed a 99% median reduction and an 88% mean reduction in the need for on-demand therapy for attacks, as well as a substantial decrease in the incidence of moderate to severe attacks.

While Andembry was generally well-tolerated, the most frequently reported adverse reactions included inflammation of the nasal passages, throat discomfort, and abdominal pain, occurring in at least 7% of patients.

Bill Mezzanotte, M.D., who leads research and development at CSL, the company behind Andembry, emphasized the significance of this approval. He noted that it offers patients living with hereditary angioedema an effective long-term management option, paired with a convenient administration method.

This advancement in treatment represents a meaningful step forward for individuals affected by hereditary angioedema, providing hope for better control of their condition and improved quality of life.