An oral pill could transform hereditary angioedema prevention

Results from the phase 3 CHAPTER-3 trial show that a once-daily oral deucrictibant extended-release (XR) tablet reduced monthly hereditary angioedema (HAE) attack rates by 83% compared to placebo — a level of efficacy that rivals injectable therapies. The drug, developed by Pharvaris, works as a bradykinin B2 receptor antagonist and was tested in 85 patients aged 12 and older across the full spectrum of HAE types, including the harder-to-treat HAE with normal C1 inhibitor — a first for any pivotal HAE trial.

Protection kicked in fast: attack reduction was maximized within the first week and sustained throughout the 24-week study. Quality of life also improved substantially, with patients reporting gains in functioning, mood, and nutrition. The safety profile was largely favorable, with most adverse events being mild to moderate.

By the Numbers

• 83% reduction in monthly HAE attack rate vs. placebo (P < .0001)
• 87% reduction in patients with HAE type 1 or 2 specifically
• 98.2% of deucrictibant patients achieved ≥50% attack rate reduction from baseline
• 65.5% achieved ≥90% reduction; 45.5% were completely attack-free
• FDA new drug application for on-demand use has a PDUFA date of April 23, 2027; prophylaxis NDA submission planned for the first half of 2027

Why it matters: HAE attacks can be life-threatening and severely impact daily life. An effective oral prophylactic option could be a game-changer for patients who struggle with or prefer to avoid injectable therapies — potentially expanding access and improving long-term adherence.