Learn how to appropriately prepare, dose, and infuse ZOLGENSMA® (onasemnogene abeparvovec-xioi).

ZOLGENSMA is a gene therapy for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA).

ZOLGENSMA has a Boxed Warning for acute serious liver injury. Please see additional Important Safety Information at the end of this video. Please also see the Full Prescribing Information at ZOLGENSMA-hcp.com.

ZOLGENSMA is a vector-based gene therapy that utilizes a type of adeno-associated virus called AAV9 to deliver a fully functional copy of the human SMN gene with a continuous promoter.

As a gene therapy, the recommended dose of ZOLGENSMA is measured in vector genomes, or vg, and is dosed based on body weight. ZOLGENSMA is provided as a kit containing 1 alcohol wipe per vial, the full Prescribing Information, a helpful quick reference guide, and 2 or more clear, single-use, 10 mL vials, containing either 5.5 mL or 8.3 mL of product, with a nominal concentration of 2.0 x 10¹³ vg/mL.

Each kit contains a unique combination of these vials, customized to meet dosing requirements based on the weight of each patient. It is best to confirm the patient’s current weight prior to treatment, so that the appropriate dose is shipped.

Before infusing ZOLGENSMA, let’s review the 5-step process for a one-time-only dose. This process is also highlighted in the ZOLGENSMA Treatment Guide, which can be found at zolgensma-hcp.com.

Step 1: Confirm diagnosis and run laboratory tests.

Prior to infusion, the prescribing physician should have:

• Confirmed a diagnosis using genetic tests for bi-allelic mutations in the SMN1 gene

• Measured the patient’s weight in kilograms to determine the appropriate dose

• Performed testing for the presence of anti-AAV9 antibodies

• And, performed baseline tests for liver function, platelet count, and troponin-I to establish reference values for each

Step 2: Store and handle ZOLGENSMA properly.

ZOLGENSMA is shipped in a special container and delivered frozen at negative 60 degrees Celsius, or negative 76 degrees Farenheit, or less. Do not refreeze. It should be refrigerated immediately upon receipt, at 2 to 8 degrees Celsius (or 36 to 46 degrees Farenheit), and protected from light.

ZOLGENSMA is stable at this temperature, for up to 14 days from receipt. ZOLGENSMA is a suspension for intravenous infusion.

ZOLGENSMA will thaw in the refrigerator and be ready for patient infusion in 12 hours. Alternatively, you can thaw ZOLGENSMA at room temperature and be ready for patient infusion in approximately 4 hours. ZOLGENSMA is a clear to slightly opaque, colorless to faint white liquid, free of particles. You should inspect ZOLGENSMA visually for particulate matter and discoloration prior to infusion. Do not use if particulates or discoloration are present. Do not shake ZOLGENSMA.

Step 3: Premedicate and plan for infusion day.

To manage potential elevations in hepatic aminotransferases, all patients should receive systemic corticosteroids, equivalent to oral prednisolone at 1 mg/kg/day, beginning 24 hours before ZOLGENSMA infusion and continuing for a minimum of 30 days.

In addition to initiating the corticosteroid regimen, ensure patients have been tested for the presence of anti-AAV9 antibodies and assessed for baseline liver function, platelet count, and troponin-I levels.

Step 4: Prepare and infuse ZOLGENSMA.

Do not use ZOLGENSMA unless it is thawed. If thawed in the refrigerator, remove ZOLGENSMA from the refrigerator on the day of dosing.

Draw up the entire quantity of all vials into a single 60 mL dosing syringe.

Next, remove the air from the syringe. Cap the syringe and deliver it to the patient treatment location at room temperature. Once the dose is drawn into the syringe, it must be used within 8 hours.

To infuse ZOLGENSMA, place a primary catheter in a peripheral vein in the arm or leg—insertion of a backup catheter is strongly recommended. As with other infusion procedures, use the appropriate guards, gowns, and aseptic technique when preparing and infusing ZOLGENSMA.

There are no specific tubing restrictions for infusing ZOLGENSMA. Program the syringe pump for saline priming or prime tubing manually with saline. When primed, attach the syringe containing ZOLGENSMA and reprogram the pump for ZOLGENSMA infusion. ZOLGENSMA should be infused slowly, over 60 minutes. At the end of the infusion, flush the line with saline.

Step 5: Monitor and postmedicate after ZOLGENSMA infusion.

After infusion, lab tests will need to be continued on a regular basis over a 3-month period or longer, as directed in the ZOLGENSMA Prescribing Information.

Monitor liver function, platelet count, and troponin-I at baseline and weekly for the first month after infusion.

Continue to monitor liver function every other week for the second and third months following infusion or longer until results are unremarkable. Continue to monitor platelet count every other week for the second and third months following infusion or longer until levels return to baseline. Monitor troponin-I once a month for the second and third months following infusion or longer until levels return to baseline.

In addition to monitoring, the daily, systemic corticosteroid treatment regimen initiated 24 hours prior to infusion will need to continue. Be certain to reinforce with caregivers that it is important to continue the daily corticosteroid dose as prescribed by the physician.

After 30 days, the recommend corticosteroid regimen varies based on the patient’s liver function assessment. For patients with unremarkable findings, the corticosteroid can be tapered over the next 28 days.

Please refer to the ZOLGENSMA Treatment Guide and Full Prescribing Information to learn more about dosing, infusing, and monitoring.

Please visit ZOLGENSMA-hcp.com for more information.