fitusiran — Medical Mutual

Initial criteria

• Patient age ≥ 12 years; AND
• Patient does not have a co-existing thrombophilic disorder or a history of, or risk factors predisposing to, thrombosis; AND
• Will not be used for the treatment of breakthrough bleeds; AND
• Patient has an antithrombin (AT) activity level of ≥ 60% prior to start of therapy and AT-activity will be monitored periodically; AND
• Patient does not have hepatic impairment (Child-Pugh Class A, B, or C); AND
• Provider will consider alternative treatments in patients with a history of symptomatic gallbladder disease, or interruption/discontinuation in patients with acute/recurrent gallbladder disease; AND
• Will NOT be used in combination with another agent used as prophylactic therapy for Hemophilia A or B (note: CFC or BPA prophylaxis discontinued by day 7 after initiating fitusiran); AND
• Diagnosis of severe Hemophilia A (FVIII <1%) or Hemophilia B (FIX ≤2%) as confirmed by coagulation testing; AND
• Must be used for routine prophylaxis to prevent or reduce the frequency of bleeding episodes; AND
• Used as primary prophylaxis in severe factor deficiency OR secondary prophylaxis in patients with ≥ 2 documented spontaneous joint bleeds; AND
• If Hemophilia A without inhibitors → must have tried and had inadequate response to emicizumab AND an antihemophilic Factor VIII prophylaxis agent unless contraindicated or not tolerated; OR
• If Hemophilia A with inhibitors → must have had previous prophylaxis therapy; OR
• If Hemophilia B without inhibitors → must have tried and had inadequate response to an antihemophilic Factor IX prophylaxis agent unless contraindicated or not tolerated; OR
• If Hemophilia B with inhibitors → must have had previous prophylaxis therapy

Reauthorization criteria

• Patient continues to meet universal and indication-specific criteria; AND
• Absence of unacceptable toxicity (e.g., severe hepatotoxicity, thromboembolic events, severe gallbladder disease); AND
• Patient has demonstrated beneficial response (bleeding frequency decreased from baseline); AND
• Latest AT-activity result guiding dosing adjustment per label: <15% → reduction in dose (discontinue if 10 mg every 2 months already); OR 15–35% → continue current dose; OR if not achieving satisfactory bleed control OR AT >35% after ≥6 months → escalate per label

Approval duration

Initial: 6 months; Renewal: 12 months