Alternate Name(s):
Zolgensma(R)
Classification:
Adeno-associated virus vector-based gene therapy
Original Date:
February 2020
Indications:
- Treatment of patients less than 2 years of age with spinal muscular dystrophy (SMA) with bi-allelic mutations in the survival motor neuron (SMN1) gene
Reconstitution and Stability:
- Supplied in 10 mL single use vials containing a volume of either 5.5 or 8.3 mL or supplied as 5 mL single use vials containing 1 mL or 5 mL.
- Stable for 14 days in the fridge once thawed.
- Thaw Zolgensma before use. The contents of the Zolgensma kit will thaw in approximately 12 hours if placed in a fridge, or in approximately 4 hours if placed at room temperature. If thawed in fridge, remove from fridge on day of dosing.
- When thawed, Zolgensma is a clear to slightly opaque, colorless to faint white liquid, free of particles. Visually inspect vial for particulate matter and discoloration prior to infusion. Do not use vials is particulate or discoloration are present.
- DO NOT SHAKE
- Draw the appropriate dose volume from all vials into a syringe, remove air from the syringe, cap the syringe, and deliver the syringe at room temperature to the patient infusion location
- Use Zolgensma within 8 hours of drawing into syringe.
- DO NOT REFREEZE
Compatibility:
- NaCL 0.9%
- No other compatibility information
Administration:
(For approved routes of administration by nursing personnel, refer to Policy for the Administration of Intravenous Medications.)
| SC | NO |
| IM | NO |
| IV Direct |
NO
|
| IV Intermittent Infusion |
YES Usual dilution: Undiluted Infusion time: 60 minutes. Administer into a peripheral limb vein (arm or leg) Flush with saline following completion of infusion |
| IV Continuous Infusion | NO |
Dosage:
(For neonatal dosages, refer to Neonatal IV Drug Manual.)
- 1.1 x 1014 vector genomes (vg)/kg
| PATIENT WEIGHT RANGE (kg) | DOSE VOLUMEamL |
| 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 patients less than 2 years of age between 2.6 and 3.5 kg b Dose volume for patients less than 2 years of age weighing greater than or equal to 13.6 kg will require a combination of Zolgensama kits |
Potential hazards of parenteral administration:
- Most common adverse reactions (>5%): elevated aminotransferases and vomiting
- Hepatic: elevated aminotransferases, acute serious liver injury
- Cardiovascular: transient increases in cardiac troponin
- Hematologic: thrombocytopenia
Notes:
- One day prior to Zolgensma infusion, begin administration of systemic corticosteroids equivalent to prednisolone at 1 mg/kg/day for a total of 30 days
- Prior to infusion, assess liver function. Continue to monitor liver function for at least 3 months after infusion.
- Monitor platelet counts before infusion, and weekly for the 1st month and then every other week for the 2nd & 3rd month until platelet counts return to baseline.
- Monitor troponin before infusion and then weekly for the 1st mnth and then monthly for the 2nd and 3rd month until troponin-1 level returns to baseline.
References:
- Zolgensma Drug Product Monograph