for hereditary angioedema (HAE) — one-time treatment
BLA· Priority Review · Orphan · RMAT
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.
About
Hereditary angioedema is a rare genetic disease that causes sudden, recurrent, and severe swelling attacks in various parts of the body. Lonvoguran zicluneran is a one-time treatment that uses CRISPR gene editing to target and modify the KLKB1 gene in liver cells. By editing this gene, the therapy aims to reduce the production of a key protein involved in swelling, potentially preventing future attacks.
