BEQVEZ is an adeno-associated virus vector-based gene therapy indicated for the treatment of adults with moderate to severe hemophilia B (congenital factor IX deficiency) who:

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Currently use factor IX prophylaxis therapy, or

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Have current or historical life-threatening hemorrhage, or

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Have repeated, serious spontaneous bleeding episodes, and,

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Do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid as detected by an FDA-approved test. (1)

For one-time single-dose intravenous infusion only. (2)

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Perform baseline testing to select patients, including testing for pre-existing antibodies to AAVRh74var, factor IX inhibitor presence, and liver health tests. (2.1)

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The recommended dose of BEQVEZ is 5 × 1011 vector genomes per kg (vg/kg) of body weight. Dose based on adjusted body weight for those with a BMI >30 kg/m2. (2.1)

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Administer BEQVEZ as an intravenous infusion after dilution in 0.9% sodium chloride with 0.25% human serum albumin (HSA) with a final volume of 200 mL over approximately 60 minutes. (2.3)

BEQVEZ is a suspension for intravenous infusion after dilution. (3)

BEQVEZ has a nominal concentration of 1 × 1013 vg/mL, and each vial contains an extractable volume of 1 mL. (3)

The total number of vials will be customized to meet dosing requirements for individual patients based on their weight. (3)

None. (4)

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Hepatotoxicity: Monitor transaminases and factor IX activity levels once or twice weekly for at least 4 months after BEQVEZ administration to mitigate the risk of potential hepatotoxicity. Consider corticosteroid treatment for transaminase elevation or a decline in factor IX activity. (2.3, 5.1)

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Infusion Reactions: Monitor during administration and for at least 3 hours after end of infusion. If symptoms occur, slow or stop administration. Restart infusion at a slower rate once reaction has resolved. (5.2)

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Malignancy: Monitor patients with risk factors for hepatocellular carcinoma (e.g., hepatitis B or C, non-alcoholic fatty liver disease, chronic alcohol consumption, non-alcoholic steatohepatitis, advanced age) with regular liver ultrasound (e.g., annually) and alpha-fetoprotein testing for 5 years following administration. In the event that a malignancy occurs after treatment with BEQVEZ, contact Pfizer Inc. at 1-800-438-1985. (5.3)

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Monitoring laboratory tests: Monitor for factor IX activity and factor IX inhibitors. (5.4)

The most common adverse reaction (incidence ≥5%) was an increase in transaminases. (6)

To report SUSPECTED ADVERSE REACTIONS, contact Pfizer Inc. at 1-800-438-1985 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

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There is limited information on the safety and effectiveness of BEQVEZ in patients with HIV infection. (8.8)

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The safety and effectiveness of BEQVEZ in patients with prior or active factor IX inhibitors have not been established. (8.9)