Usual Adult Dose for Hemophilia B

Usual dose: 2 x 10(13) genome copies per kilogram of body weight (or 2 mL/kg body weight) as a single IV infusion

Comments:

• Refer to manufacturer product information for detailed dose calculation.
• Patients with any of the following criteria are eligible for this treatment:
• Currently on Factor IX prophylaxis therapy
• Have current or historical life-threatening hemorrhage
• Have repeated, serious spontaneous bleeding episodes
• The following tests should be performed for screening patients before treatment:
• Factor IX inhibitor titer testing: A re-test should be performed within 2 weeks in case of initial positive test results. If both tests are positive, this therapy should not be administered.
• Liver health assessments: Enzyme testing (alanine aminotransferase, aspartate aminotransferase, alkaline phosphatase and total bilirubin), hepatic ultrasound and elastography
• Consult hepatologist in case of radiological liver abnormalities and/or sustained liver enzyme elevations to assess appropriateness of this treatment.

Renal Dose Adjustments

Data not available

Liver Dose Adjustments

Data not available

Dose Adjustments

Data not available

Precautions

CONTRAINDICATIONS: None

Safety and efficacy have not been established in patients younger than 18 years.

Consult WARNINGS section for additional precautions.

Dialysis

Data not available

Other Comments

Administration advice:

• This drug should be administered as a single-dose intravenous infusion through peripheral venous catheter at a constant infusion rate of 500 mL/hour.
• Diluted preparation should be visually inspected prior to administration; It should be clear and colorless.
• The diluted product should be administered within 24 hours of preparation.
• An integrated (in-line) polyethersulfone (PES) 0.2 mcm filter should be used.
• This product should not be administered as an intravenous push or bolus.
• A central line or port should not be used.
• Do not infuse this product in the same intravenous line with other products.
• If an infusion reaction occurs during administration, the infusion rate can be reduced or stopped, and restarted at a slower rate when resolved.
• Flush the intravenous line with saline after the infusion is complete to ensure complete delivery of the drug product.
• In case of surface spilling, treat with virucidal agent with proven activity against non-enveloped viruses.
• Properly dispose the unused drug product as per local biosafety guidelines.
• Refer to manufacturer product information for other detailed instructions.

• Store at 2C to 8C (36F to 46F); do not freeze.
• Store in original carton until use.
• Protect from light until preparation and use.
• Store the diluted preparation in the infusion bag at 15C to 25C (59F to 77F) away from light.

• Refer to manufacturer product labeling for detailed instructions.

• This drug product is only compatible with intravenous 0.9% saline. Do not mix this with any other product.

• Monitor liver enzyme and Factor IX activity following administration of this drug product.
• Refer to manufacturer product labeling for detailed information on testing.

• Patients should be tested for Factor IX inhibitors before the administration of this drug product. If the test is positive, the patient is not a good candidate for this therapy.
• A liver ultrasound and elastography should be performed prior to the administration of this drug product. Patients with pre-existing hepatocellular carcinoma should be monitored annually for 5 years following infusion of this drug product.
• This drug product may cause infusion reactions. Patients are monitored for at least 3 hours post infusion. In the event of an infusion reaction, the infusion rate can be reduced or stopped and resumed at a slower rate once resolved.
• This drug product can elevate liver enzymes. Patients are tested weekly to monitor liver enzymes for 3 months post administration of this drug product. Corticosteroids can be given to manage these events.
• In the event of uncontrolled bleeding, or if bleeding resumes after the administration of this drug product, blood tests should be performed for Factor IX activity and neutralizing Factor IX inhibitors.
• Patients should not donate blood, organs, tissues, or cells for transplantation due to vector distribution in blood (within the body), and vector shedding in semen and other excreta and secreta after administration.
• Patients who intend to receive treatment with this drug product are encouraged to enroll in a study to measure pre-existing anti-AAV5 neutralizing antibodies by calling CSL Behring at 1-800-504-5434. The study evaluates the effect of pre-existing anti-AAV5 neutralizing antibodies on the risk of bleeding.