A single-dose intravenous gene therapy used to treat adults with severe Hemophilia B.
HEMGENIX is indicated for treatment of adults with Hemophilia B (congenital Factor IX deficiency) who: Currently use Factor IX prophylaxis therapy, or Have current or historical life-threatening hemorrhage, or Have repeated, serious spontaneous bleeding episodes. HEMGENIX is an adeno-associated virus vector-based gene therapy indicated for the treatment of adults with Hemophilia B (congenital Factor IX deficiency) who: Currently use Factor IX prophylaxis therapy, or Have current or historical life-threatening hemorrhage, or Have repeated, serious spontaneous bleeding episodes.
12.1 Mechanism of Action HEMGENIX is an adeno-associated virus serotype 5 (AAV5) based gene therapy designed to deliver a copy of a gene encoding the Padua variant of human coagulation Factor IX (hFIX-Padua). Single intravenous infusion of HEMGENIX results in cell transduction and increase in circulating Factor IX activity in patients with Hemophilia B.
Sources: drug label, approval and manufacturer data from the U.S. FDA via the openFDA API; shortage status from the FDA Drug Shortage Database. This page is a plain-English summary for general information and is not medical advice. It is not exhaustive and may not reflect the latest label — always consult the official prescribing information and your clinician or pharmacist. Brand availability, indications and approvals differ between the US, EU/Ireland (EMA/HPRA) and other regions.