About Onasemnogene Abeparvovec-xioi
Class: | Gene therapy, specifically an adeno-associated virus (AAV) vector-based therapy
Use: | Treatment of spinal muscular atrophy (SMA) in pediatric patients less than 2 years of age with bi-allelic mutations in the SMN1 gene
Adult dose: | Not applicable; ZOLGENSMA is not indicated for adults
Pediatric dose: | One-time intravenous infusion of 2 x 10^13 vector genomes/mL
Side effects: | Common side effects include elevated liver enzymes (ALT, AST), vomiting, diarrhea, and respiratory i
Drug Class
Gene therapy, specifically an adeno-associated virus (AAV) vector-based therapy
Uses & Indications
Treatment of spinal muscular atrophy (SMA) in pediatric patients less than 2 years of age with bi-allelic mutations in the SMN1 gene
Storage Requirements
Store in the original carton at -80°C to -60°C (-112°F to -76°F); do not freeze or shake the vial
Manufacturer & Packaging
Manufacturer: AveXis Inc., United States of America
Package Size
Kit (5.5mL vial x X + 8.3mL vial x X)
Price & Supplier
Price in UAE: AED 8351437.50
Dosage Information
Adult Dose
Not applicable; ZOLGENSMA is not indicated for adults
Pediatric Dose
One-time intravenous infusion of 2 x 10^13 vector genomes/mL
Side Effects
Common side effects include elevated liver enzymes (ALT, AST), vomiting, diarrhea, and respiratory infections
Contraindications & Precautions
Contraindicated in patients with a history of serious hypersensitivity reactions to the drug or any of its components
Important Warnings
Risk of acute liver failure; monitor liver function tests prior to and after administration; potential for immunogenicity leading to neutralizing antibodies against AAV9, which may affect efficacy of future AAV-based therapies
Medical Disclaimer
This information is for educational purposes only and should not replace professional medical advice.
Always consult with a licensed healthcare provider or pharmacist in the UAE before taking any medication.
The information presented here is specific to medicines available in UAE pharmacies and is regularly updated
to ensure accuracy for patients and healthcare professionals in the United Arab Emirates.