for hereditary angioedema
Sponsor announced submission . 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 is a rare genetic disorder that causes sudden, severe, and painful swelling attacks in various parts of the body, often beginning in childhood. Lonvoguran ziclumeran is a CRISPR-based gene-editing therapy designed to be delivered as a one-time intravenous infusion. By editing the DNA in liver cells, this treatment aims to provide a durable, functional cure by enabling the body to produce the protein it lacks, potentially preventing future swelling attacks.