Skip to main content

Zolgensma FDA Approval History

Last updated by Judith Stewart, BPharm on July 1, 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.
1. AveXis Announces Innovative Zolgensma® Gene Therapy Access Programs for US Payers and Families - Accessed at - May 29, 2019

Development timeline for Zolgensma

Aug  6, 2019FDA Statement on Data Accuracy Issues with Recently Approved Gene Therapy
May 26, 2019Approval FDA Approves Zolgensma (onasemnogene abeparvovec-xioi) Gene Therapy to Treat Pediatric Patients with Spinal Muscular Atrophy
May  5, 2019AveXis Presented Robust Data at AAN Demonstrating Efficacy of Zolgensma in Broad Spectrum of Spinal Muscular Atrophy (SMA) Patients
Apr 16, 2019AveXis Data Reinforce Effectiveness of Zolgensma in Treating Spinal Muscular Atrophy (SMA) Type 1
Dec  3, 2018Novartis Announces FDA Filing Acceptance and Priority Review of AVXS-101, a One-Time Treatment Designed to Address the Genetic Root Cause of SMA Type 1

Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.