Etranacogene dezaparvovec, sold under the brand name Hemgenix is a gene therapy used for the treatment of hemophilia B.
[7][8] Etranacogene dezaparvovec was approved for medical use in the United States in November 2022,[6][7][9] in the European Union in February 2023.
[2] Etranacogene dezaparvovec is indicated for the treatment of adults with hemophilia B (congenital Factor IX deficiency) who use Factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.
[7] Effectiveness was established based on decreases in the men's annualized bleeding rate (ABR).
[7] The FDA granted the application for etranacogene dezaparvovec priority review, orphan drug, and breakthrough therapy designations.