Onasemnogene Abeparvovec Application Scope

Onasemnogene abeparvovec, marketed as Zolgensma, is a gene therapy indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.

Onasemnogene Abeparvovec Characteristics

Ingredients:
- Active Ingredient: Onasemnogene abeparvovec-xioi
- Inactive Ingredients: Tromethamine, magnesium chloride, sodium chloride, poloxamer 188, hydrochloric acid (for pH adjustment), and water for injections.
Properties:
- Onasemnogene abeparvovec is a recombinant adeno-associated virus vector-based gene therapy designed to deliver a functional copy of the SMN1 gene to motor neuron cells.
Specification:
- Each vial contains a suspension with a concentration of 2.0 × 10^13 vector genomes per milliliter (vg/mL).
Packaging Specification:
- Supplied in 10 mL vials with fill volumes of either 5.5 mL or 8.3 mL. The product is provided as a customized kit containing 2 to 14 vials, depending on the patient’s weight.
Storage:
- Store and transport frozen at temperatures ≤ -60°C. Upon receipt, immediately place the kit in a refrigerator at 2°C to 8°C. The product is stable for 14 days from receipt when stored at this temperature. Do not refreeze.
Expiry Date:
- The shelf life is 24 months when stored as recommended.
Executive Standard:
- Approved by the U.S. Food and Drug Administration (FDA) under Biologics License Application (BLA) number 125694.
- Approval Number:
- BLA 125694.
Date of Revision:
- Refer to the latest prescribing information for the most current revision date.
Manufacturer:
- Novartis Gene Therapies, Inc.

Guidelines for the Use of Onasemnogene Abeparvovec

Dosage and Administration:
- Administer as a single-dose intravenous infusion over 60 minutes. The recommended dose is determined based on the patient’s weight. Prior to infusion, initiate systemic corticosteroid therapy (e.g., prednisolone at 1 mg/kg/day) one day before administration and continue for at least 30 days to mitigate potential hepatotoxicity. Monitor liver function for at least 3 months after infusion.Adverse Reactions:
- Common adverse reactions include elevated liver enzymes (aminotransferases), vomiting, and thrombocytopenia. Serious liver injury and acute liver failure have been reported. Monitor liver function, platelet counts, and troponin-I levels before and after infusion.
- Contraindications:
- No specific contraindications have been identified.
Precautions:
- Due to potential hepatotoxicity, administer systemic corticosteroids before and after infusion and monitor liver function closely. Adjust vaccination schedules to accommodate corticosteroid use, as live vaccines may be contraindicated during immunosuppressive therapy. Monitor for thrombocytopenia and elevated troponin-I levels. Temporary vector shedding may occur; follow proper hygiene procedures when handling patient waste for one month after infusion.

Onasemnogene Abeparvovec Interactions

Drug Interactions:
- Concomitant use of corticosteroids may necessitate adjustments to vaccination schedules, particularly with live vaccines, which may be contraindicated during immunosuppressive therapy. Consult vaccination guidelines for immunocompromised patients.

Note:

If there is a new packaging for the drug, the new packaging shall prevail. The above information is sourced from DengYue Medicine.
It is only for internal discussion among medical staff and does not serve as a basis for medication. For specific medication guidelines, please consult the attending physician.