Hemgenix (etranacogene dezaparvovec-drlb) — Medica

Initial criteria

• Patient is male
• age ≥ 18 years
• Patient has not received a gene therapy for hemophilia B in the past (no claim for Hemgenix or Beqvez, or physician confirmation if unavailable)
• Patient has moderately severe or severe hemophilia B with baseline Factor IX level ≤ 2% of normal [documentation required]
• Patient meets ONE of the following: (i) history of use of Factor IX therapy for ≥ 150 exposure days; OR (ii) history of life-threatening hemorrhage AND on-demand Factor IX therapy required; OR (iii) history of repeated, serious spontaneous bleeding episodes AND on-demand Factor IX therapy required
• Factor IX inhibitor titer testing performed within 30 days and patient is negative for inhibitors [documentation required]
• Patient does not have active infection with hepatitis B or hepatitis C virus AND is not currently receiving antiviral therapy for prior hepatitis B/C exposure [documentation required]
• Patient does not have uncontrolled HIV infection
• Liver function testing within 30 days shows: alanine aminotransferase ≤ 2× ULN, aspartate aminotransferase ≤ 2× ULN, total bilirubin ≤ 2× ULN, and alkaline phosphatase ≤ 2× ULN [documentation required]
• No evidence of advanced liver impairment and/or advanced fibrosis
• Platelet count ≥ 50 × 10^9/L within 30 days [documentation required]
• Within 30 days, patient has estimated creatinine clearance ≥ 30 mL/min OR creatinine level ≤ 2× ULN [documentation required]
• Medication prescribed by a hemophilia specialist physician
• Current body weight obtained within 30 days [documentation required]
• If all above criteria are met, approve one dose to provide a one-time (per lifetime) single IV infusion of 2 × 10^13 genome copies/kg [verification required]

Approval duration

one-time (per lifetime)