Lonvoguran Ziclumeran Lowers Hereditary Angioedema Attacks
Key Takeaways
- A single infusion of lonvoguran ziclumeran was associated with a substantially lower monthly hereditary angioedema attack rate than placebo.
- The phase 3, double-blind trial randomized 80 patients in a 2:1 ratio to one 50-mg intravenous dose of lonvoguran ziclumeran or placebo, with attacks per month from week 5 through week 28 as the primary end point.
- Adverse events were frequent in both groups, and no serious or grade 3 or higher adverse events were reported in the lonvoguran ziclumeran group during the reported safety assessment window.
In the phase 3, double-blind trial, patients 16 years of age or older with hereditary angioedema and C1 inhibitor deficiency were randomized in a 2:1 ratio, with 52 assigned to lonvoguran ziclumeran and 28 assigned to placebo. Treatment consisted of a single intravenous infusion of lonvoguran ziclumeran 50 mg or placebo. The prespecified primary end point was the number of investigator-confirmed hereditary angioedema attacks per month from week 5 through week 28 after infusion.
As of February 10, 2026, the median follow-up was 7.5 months, with a range of 4.9 to 12.8 months. From weeks 5 through 28, the least-squares mean monthly attack rate was 0.26 with lonvoguran ziclumeran and 2.10 with placebo. The corresponding 95% confidence intervals were 0.15 to 0.45 and 1.55 to 2.86, respectively. The relative difference was -87%, with a 95% confidence interval of -93 to -78 and a P value below 0.001.
Adverse events during or after infusion were reported in 92% of the lonvoguran ziclumeran group and 86% of the placebo group from day 1 through week 28. In the lonvoguran ziclumeran group, the most common adverse events were infusion-related reaction, headache, fatigue, back pain, and upper respiratory tract infection. No serious adverse events or grade 3 or higher adverse events were reported in the lonvoguran ziclumeran group.