Zolgensma Approval History
Reviewed by J.Stewart BPharm Last updated on May 29, 2019.
FDA Approved: Yes (First approved May 24, 2019)
Brand name: Zolgensma
Generic name: onasemnogene abeparvovec-xioi
Dosage form: Suspension for Intravenous Infusion
Company: Novartis Pharmaceuticals Corporation
Treatment for: Spinal Muscular Atrophy
Zolgensma (onasemnogene abeparvovec-xioi) is an adeno-associated virus vector-based gene therapy indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA).
- SMA is a rare, genetic neuromuscular disease caused by a defective or missing survival motor neuron 1 (SMN1) gene. Infants who do not have a functional SMN1 gene lose the motor neurons responsible for muscle functions such as breathing, swallowing, speaking and walking. In its most severe form, SMA can lead to permanent ventilation or death by age 2.
- Zolgensma works to halt disease progression by replacing the defective or missing SMN1 gene. It is administered as a single, one-time infusion.
- Zolgensma carries a boxed warning that acute serious liver injury can occur. The most common side effects include elevated liver enzymes and vomiting.
- At the time of approval, the cost of Zolgensma was $2.125 million1, making it the world's most expensive drug.
Development History and FDA Approval Process for Zolgensma
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