for hereditary angioedema (HAE) — one-time treatment
Sponsor announced submission APR 27 2026. FDA filing review (~60 days) in progress; PDUFA date estimated as acceptance + 10 months Standard Review. Card updates with confirmed dates when FDA acceptance announcement lands.
Hereditary angioedema (HAE) causes sudden, severe swelling attacks that can block airways or distort limbs without warning, often starting in childhood. Lonvoguran ziclumeran uses CRISPR gene editing to permanently disable the KLKB1 gene in liver cells, reducing production of the kallikrein protein that triggers these dangerous swelling episodes. By editing this genetic root cause, the one-time treatment could prevent future attacks without requiring lifelong medication.