Hemophilia – Hemgenix®

Hemgenix is covered as a one-time (per lifetime) IV gene therapy dose (2×10^13 genome copies/kg) for adult males (≥18) with moderately severe or severe hemophilia B (baseline Factor IX ≤2%) and is excluded for prior hemophilia B gene‑therapy recipients, patients with active hepatitis B/C or on antiviral therapy, uncontrolled HIV, prior Factor IX inhibitors, advanced liver impairment/fibrosis, or failure to meet required lab/clinical criteria. Approval requires a hemophilia specialist prescriber and documentation of baseline Factor IX ≤2%, either ≥150 Factor IX exposure days or qualifying bleeding history, negative Factor IX inhibitor test within 30 days, LFTs (ALT/AST/alk phos/total bilirubin) ≤2× ULN, platelets ≥50×10^9/L, renal function (CrCl ≥30 mL/min or creatinine ≤2× ULN), recent body weight for dosing, and verification of no prior gene therapy in claims or by physician.

"FDA-indicated 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..."