Hemgenix (etranacogene dezaparvovec-drlb)

Hemgenix is authorized as a one‑time (lifetime) gene therapy for adult males (≥18) with congenital hemophilia B and baseline Factor IX ≤2%, and is excluded for females, patients <18, those with prior hemophilia B gene therapy, active HBV/HCV or uncontrolled HIV, positive FIX inhibitors, advanced liver disease, platelet <50×10^9/L, or inadequate renal function. Approval requires documentation (within 30 days) of baseline FIX level and negative inhibitor titer, LFTs ≤2×ULN, platelet ≥50×10^9/L, either creatinine clearance ≥30 mL/min or creatinine ≤2×ULN, current weight, hemophilia specialist prescribing, qualifying bleeding history or ≥150 FIX exposure days, confirmation of no prior gene therapy, and a plan to stop routine FIX prophylaxis after adequate post‑treatment FIX levels (single IV, weight‑based infusion).

"Must be prescribed by a hemophilia specialist physician (lack of such specialty may preclude approval)."