Select a medication above to begin.
Zolgensma (onasemnogene abeparvovec-xioi)
onasemnogene abeparvovec
Black Box Warnings .
Acute Serious Liver Injury and Failure
fatal cases of acute liver failure reported; acute serious liver injury and elevated ALT/AST may occur; patients with pre-existing liver impairment may be at higher risk; assess LFTs at baseline, then continue monitoring for at least 3mo after infusion and as clinically indicated; administer systemic corticosteroid to all patients before and after infusion
Adult Dosing .
Adult dosing is currently unavailable or not applicable for this drug.
Peds Dosing .
- Dosage forms: INJ
Special Note
- [drug name clarification]
- Info: nonproprietary name = onasemnogene abeparvovec-xioi
spinal muscular atrophy
- [<2 yo]
- Dose: 1.1 x10^14 vector genomes/kg/dose IV x1; Info: for patients with biallelic mutations in SMN1 gene; use with oral corticosteroid, see pkg insert for dose and timing
renal dosing
- [not defined]
- renal impairment: not defined
- HD/PD: not defined
hepatic dosing
- [see below]
- hepatic impairment: not defined, caution advised
Contraindications / Cautions .
- hypersensitivity to drug or ingredient
- infection, active
- caution: hepatic impairment
Drug Interactions .
Overview
onasemnogene abeparvovec
SMN1 gene replacement
-
None
No significant interactions known or found for this drug. Caution always advised with multiple medications.
Adverse Reactions .
Serious Reactions
- hepatic injury, acute
- hepatic failure, acute
- thrombocytopenia
- thrombotic microangiopathy
- tumorigenicity risk
- cardiotoxicity (animal studies)
Common Reactions
- ALT or AST elevated
- thrombocytopenia
- vomiting
- troponin-I elevated
Safety/Monitoring .
Monitoring Parameters
CBC, Cr, PTT, serum albumin at baseline; ALT, AST, PT/INR, total bilirubin at baseline, then qwk x2mo and until corticosteroid taper completed, then q2wk x1mo; Plt at baseline, then at least qwk x1mo, then q2wk x2mo and until normalization; troponin-I at baseline, then qwk x1mo, then qmo x2mo
Pregnancy/Lactation .
Pregnancy
Clinical Summary
N/A; drug unlikely to be used during pregnancy based on indication
Lactation
Clinical Summary
N/A; drug unlikely to be used while breastfeeding based on indication
Pharmacology .
Metabolism: for onasemnogene abeparvovec: unknown; CYP450: unknown
Excretion: for onasemnogene abeparvovec: feces, saliva, urine; Half-life: unknown
Subclass: Motor Neuron Diseases
Mechanism of Action
for onasemnogene abeparvovec: recombinant adeno-associated viral vector delivers functional copy of survival motor neuron 1 (SMN1) gene to target motor neurons, improving muscle movement
Formulary .
No Formulary Selected
Manufacturer/Pricing .
Manufacturer: Novartis Gene Therapies, Inc.
DEA/FDA: Rx
Retail Price
Estimated prices only. For current pricing, visit GoodRx.
intravenous kit:
- 20 trillion vg/mL (1 kit, 1 (5.5 mL) and 5 (8.3 mL) vials): $2,167,510.00
intravenous kit:
- 20 trillion vg/mL (1 kit, 1 (5.5 mL) and 7 (8.3 mL) vials): $2,167,510.00
intravenous kit:
- 20 trillion vg/mL (1 kit, 2 (5.5 mL) and 4 (8.3 mL) vials): $2,167,510.00
intravenous kit:
- 20 trillion vg/mL (1 kit, 2 (5.5 mL) and 7 (8.3 mL) vials): $2,167,510.00
intravenous kit:
- 20 trillion vg/mL (1 kit, 2 (8.3 mL) vials): $2,167,510.00
intravenous kit:
- 20 trillion vg/mL (1 kit, 5 (8.3 mL) vials): $2,167,510.00
intravenous kit:
- 20 trillion vg/mL (1 kit, 8 (8.3 mL) vials): $2,167,510.00
intravenous kit:
- 20 trillion vg/mL (1 kit, 4 (8.3 mL) vials): $2,167,510.00
intravenous kit:
- 20 trillion vg/mL (1 kit, 2 (5.5 mL) and 1 (8.3 mL) vials): $2,167,510.00
intravenous kit:
- 20 trillion vg/mL (1 kit, 2 (5.5 mL) and 6 (8.3 mL) vials): $2,167,510.00
intravenous kit:
- 20 trillion vg/mL (1 kit, 1 (5.5 mL) and 3 (8.3 mL) vials): $2,167,510.00
intravenous kit:
- 20 trillion vg/mL (1 kit, 1 (5.5 mL) and 4 (8.3 mL) vials): $2,167,510.00
intravenous kit:
- 20 trillion vg/mL (1 kit, 2 (5.5 mL) and 2 (8.3 mL) vials): $2,167,510.00
intravenous kit:
- 20 trillion vg/mL (1 kit, 9 (8.3 mL) vials): $2,167,510.00
intravenous kit:
- 20 trillion vg/mL (1 kit, 1 (5.5 mL) and 6 (8.3 mL) vials): $2,167,510.00
intravenous kit:
- 20 trillion vg/mL (1 kit, 2 (5.5 mL) and 3 (8.3 mL) vials): $2,167,510.00
intravenous kit:
- 20 trillion vg/mL (1 kit, 2 (5.5 mL) and 5 (8.3 mL) vials): $2,167,510.00
intravenous kit:
- 20 trillion vg/mL (1 kit, 3 (8.3 mL) vials): $2,167,510.00
intravenous kit:
- 20 trillion vg/mL (1 kit, 6 (8.3 mL) vials): $2,167,510.00
intravenous kit:
- 20 trillion vg/mL (1 kit, 1 (5.5 mL) and 8 (8.3 mL) vials): $2,167,510.00
intravenous kit:
- 20 trillion vg/mL (1 kit, 1 (5.5 mL) and 2 (8.3 mL) vials): $2,167,510.00
intravenous kit:
- 20 trillion vg/mL (1 kit, 7 (8.3 mL) vials): $2,167,510.00
Join Now to View Patient Handouts!
Create a FREE epocrates Online account to access patient medication instructions. Your patients and caregivers will appreciate you printing these friendly handouts, available in English and Spanish. Each handout addresses common concerns such as how to take the medication, and possible side effects.
Current Members: Sign In.
Pill Pictures
Join Now to View Pill Pictures!
Create a FREE epocrates Online account to access full-color images of medications within the drug monograph.
Current Members: Sign In.