Qfitlia™
Drug - Qfitlia™ (fitusiran) [Genzyme Corporation]
January 2026
Therapeutic Area - Hemophilia treatments
Initial approval criteria
- Patient is ≥ 12 years of age; AND
- Patient has a diagnosis of severe hemophilia A (congenital factor VIII deficiency) or hemophilia B (congenital factor IX deficiency; also known as Christmas Disease)
- Must be used for routine prophylaxis to prevent or reduce the frequency of bleeding episodes; AND
- Used as treatment in one of the following:
- Primary prophylaxis in patients with severe factor deficiency; OR
- Secondary prophylaxis in patients with at least ONE documented episode of spontaneous bleeding; AND
- Will NOT be used for the treatment of breakthrough bleeds (Note: on-demand clotting factor concentrates [CFC] or bypassing agents [BPA] may be administered, with a reduced dose and frequency when occurring > 7 days after initiation of Qfitlia, on an as-needed basis for the treatment of breakthrough bleeds in patients being treated with Qfitlia); AND
- Patient has an antithrombin (AT) activity level of ≥ 60% prior to start of therapy and AT activity will be monitored periodically, as outlined in the prescribing information, throughout therapy; AND
- Patient does NOT have a co-existing thrombophilic disorder or a history of, or risk factors predisposing to, thrombosis; AND
- Provider will consider alternative treatments in patients with a history of symptomatic gallbladder disease, or interruption/discontinuation of therapy in patients with acute/recurrent gallbladder disease; AND
- Patient does NOT have hepatic impairment (Child-Pugh Class A, B and C); AND
- Will NOT be used in combination with any of the following (Note: patient may continue their prior CFC or BPA prophylaxis for the first 7 days of Qfitlia treatment; discontinue CFC or BPA prophylaxis no later than 7 days after the initial dose of Qfitlia):
- Hemophilia BPA prophylaxis (e.g., factor VIIa, anti-inhibitor coagulant complex); OR
- Immune tolerance induction with clotting factor products (e.g., factor VIII or factor IX concentrates) as prophylactic therapy; OR
- Emicizumab-kxwh (Hemlibra) for hemophilia A with inhibitors.
- Initial approval is for 6 months
Renewal criteria
- Patient must continue to meet the above criteria; AND
- Patient has demonstrated a beneficial response to therapy (e.g., the frequency of bleeding episodes has decreased from pre-treatment baseline); AND
- Patient’s latest AT activity result is categorized as one of the following:*
- Less than 15%; AND
- Reduction in dose according to package labeling (Note: patient is already receiving 10 mg every 2 months must discontinue therapy); OR
- 15% to 35%; AND
- Continue at the current dosage; OR
- Less than 15%; AND
- Patient has NOT achieved satisfactory bleed control compared to baseline or the patient’s latest AT activity result is categorized as > 35% after ≥ 6 months*; AND
- Escalation in dose and frequency according to package labeling; AND
- Absence of unacceptable toxicity from the drug (e.g., severe hepatotoxicity, thromboembolic events, severe gallbladder disease).
- Renewal approval is for 12 months
* Note: Patient AT activity should be monitored at prescribed times following the initiation of therapy and after any dose modifications using an FDA-cleared test
Quantity limits
- 50 mg every month
Questions
Provider Call Center: (844) 575-7887