Etranacogene Dezaparvovec (Hemgenix)

Abstract

CADTH recommends that public drug plans reimburse Hemgenix for the treatment of hemophilia B if certain conditions are met. Hemgenix should only be covered to treat patients (≥ 18 years of age) with moderately severe to severe hemophilia B (circulating coagulation factor IX [FIX] ≤ 2%) if their bleeding requires ongoing prophylactic treatment, their titre of the neutralizing antibody to variant adeno-associated virus 5 (AAV5) is below 1:900, they do not have FIX inhibitors, and if they have not previously received gene therapy to treat hemophilia B. Hemgenix should only be reimbursed if it is prescribed by specialists who are experts in treating hemophilia B and the cost of Hemgenix is reduced.