ose and Administration
The recommended dose of ZOLGENSMA is 1.1 × 1014 vector genomes per kilogram (vg/kg) ofbody weight.
Table 1: Dosing
Patient Weight Range (kg) Dose Volumea
(mL)
2.6 – 3.0 16.5
3.1 – 3.5 19.3
3.6 – 4.0 22.0
4.1 – 4.5 24.8
4.6 – 5.0 27.5
5.1 – 5.5 30.3
5.6 – 6.0 33.0
6.1 – 6.5 35.8
6.6 – 7.0 38.5
7.1 – 7.5 41.3
7.6 – 8.0 44.0
8.1 – 8.5 46.8
8.6 – 9.0 49.5
9.1 – 9.5 52.3
9.6 – 10.0 55.0
10.1 – 10.5 57.8
10.6 – 11.0 60.5
11.1 – 11.5 63.3
11.6 – 12.0 66.0
12.1 – 12.5 68.8
12.6 – 13.0 71.5
13.1 – 13.5b 74.3
a Dose volume is calculated using the upper limit of the patient weight range for pediatric patients less than 2 yearsof age between 2.6 kg and 13.5 kg
b Dose volume for pediatric patients less than 2 years of age weighing equal to or greater than 13.6 kg will require acombination of ZOLGENSMA kits.
• Prior to ZOLGENSMA infusion
o Assess liver function [see Boxed Warning, Laboratory Testing and Monitoring toAssess Safety (2.3), Warnings and Precautions (5.1) and Use in SpecificPopulations (8.6)].
o Measure platelet counts and troponin-I [see Laboratory Testing and Monitoring toAssess Safety (2.3), Warnings and Precautions (5.2)(5.3)]
o Perform baseline testing for the presence of anti-AAV9 antibodies [seeLaboratory Testing and Monitoring to Assess Safety (2.3), Adverse Reactions(6.2)].
• One day prior to ZOLGENSMA infusion, begin administration of systemiccorticosteroids equivalent to oral prednisolone at 1 milligram per kilogram of bodyweight per day (mg/kg/day) for a total of 30 days.
• Administer ZOLGENSMA as a single-dose intravenous infusion through a venouscatheter.
Follow the steps below for infusion:
1. Place a primary catheter into a vein (generally a peripheral vein in the arm or leg).
Insertion of a back-up catheter is recommended.
2. Program syringe pump for saline priming, or prime tubing manually with saline.
3. Administer ZOLGENSMA as a slow infusion over 60 minutes. DO NOT INFUSE ASAN INTRAVENOUS PUSH OR BOLUS.
4. Flush line with saline following completion of infusion.
• Monitor liver function by clinical examination and by laboratory testing on a regularbasis [see Laboratory Testing and Monitoring to Assess Safety (2.3)].
o At the end of the 30-day period of systemic corticosteroid treatment, check liverstatus clinically and by assessing ALT, AST, total bilirubin, and prothrombintime.
o For patients with unremarkable findings (normal clinical exam, total bilirubin, andprothrombin time, and ALT and AST levels below 2 × upper limit of normal
(ULN)), taper the corticosteroid dose over the next 28 days [see Warnings andPrecautions (5.1)].
o If liver function abnormalities persist, continue systemic corticosteroids(equivalent to oral prednisolone at 1 mg/kg/day) until AST and ALT values areboth below 2 × ULN and all other assessments return to normal range, and thentaper the corticosteroid dose over the next 28 days.
o Consult expert(s) if patients do not respond adequately to the equivalent of
1 mg/kg/day oral prednisolone.
2.2 Preparation
• Thaw ZOLGENSMA before use. The contents of the ZOLGENSMA kit will thaw inapproximately 12 hours if placed in a refrigerator, or in approximately 4 hours ifplaced at room temper |
