Beqvez (fidanacogene elaparvovec-dzkt) — Medica

Initial criteria

• Patient is male
• age ≥ 18 years
• Patient has not received a gene therapy for hemophilia B in the past [verification in claims history required]
• Patient has moderately severe or severe hemophilia B as evidenced by baseline (without Factor IX replacement therapy) Factor IX level ≤ 2% of normal [documentation required]
• Patient meets ONE of the following:
• - According to the prescribing physician, the patient has a history of use of Factor IX therapy for ≥ 150 exposure days; OR
• - Patient has a history of life-threatening hemorrhage AND on-demand use of Factor IX therapy was required for this life-threatening hemorrhage; OR
• - Patient has a history of repeated, serious spontaneous bleeding episodes AND on-demand use of Factor IX therapy was required for these serious spontaneous bleeding episodes
• Patient does not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid by an approved test [documentation required]
• Factor IX inhibitor titer testing has been performed within 30 days and patient is negative for Factor IX inhibitors [documentation required]
• Patient does not have an active infection with hepatitis B virus or hepatitis C virus [documentation required]
• Patient is not currently receiving antiviral therapy for a prior hepatitis B virus or hepatitis C virus exposure [documentation required]
• According to the prescribing physician, patient does not have uncontrolled human immunodeficiency virus infection
• Patient has undergone liver function testing within 30 days and meets ALL: alanine aminotransferase ≤ 2 × ULN, aspartate aminotransferase ≤ 2 × ULN, total bilirubin ≤ 1.5 × ULN, alkaline phosphatase ≤ 2 × ULN [documentation required]
• Patient does not have evidence of advanced liver impairment and/or advanced fibrosis
• Within 30 days, platelet count ≥ 100 x 10⁹/L [documentation required]
• Within 30 days, creatinine ≤ 2.0 mg/dL [documentation required]
• Prescribed by a hemophilia specialist physician
• Current patient body weight obtained within 30 days [documentation required]
• If criteria met, approve one-time (per lifetime) single dose of 5 × 10¹¹ vector genomes per kg of body weight by intravenous infusion [verification required]
• Dose based on adjusted body weight if body mass index > 30 kg/m²

Approval duration

one-time (per lifetime) single dose