Zolgensma
A one-time gene therapy for spinal muscular atrophy in infants.
Where Onasemnogene Abeparvovec is approved
All regulators →Of the three regulators tracked here, the first to approve Onasemnogene Abeparvovec was the EU, on 18 May 2020. It is approved in 2 of the 3 regulators tracked here.
- 2 EU safety letters (DHPC), latest: adverse event for Zolgensma, 16 Feb 2023
From the regulators' own registers: FDA Drugs@FDA, the EMA medicines list and Health Canada's Notice of Compliance database. Dates are the first listed approval of a product containing only this substance; combination products and nationally authorised EU medicines are not counted.
Onasemnogene Abeparvovec patents and exclusivity
Expiry tracker →Protections still running for marketed products containing Onasemnogene Abeparvovec, as listed by the regulators. Expired patents and exclusivities, and discontinued products, are not shown.
- Zolgensma for treatment of spinal muscular atrophy: until 18 May 2030. Union Register
Reproduced from the FDA Orange Book and Purple Book (checked 5 Oct 2026) and the European Commission's Union Register of medicinal products (CC BY 4.0; the EU expiry date is calculated exactly as the Commission's register displays it). Listed patents and dates are as submitted to or published by the regulator and may not reflect litigation, invalidation, settlements, lapsed fees or later extensions. This is not legal advice or a freedom-to-operate opinion, and it does not say when a generic or biosimilar will launch.
How Onasemnogene Abeparvovec works
All drug targets →- Survival motor neuron protein exogenous gene Exogenous geneTarget: Survival motor neuron proteinChEMBL only
- Proximal spinal muscular atrophyPhase 3
- Metabolic dysfunction-associated steatohepatitisPhase 1
- Non-alcoholic fatty liverPhase 1
- Spinal muscular atrophy, type 1Phase 1
Mechanisms and diseases from ChEMBL via the Open Targets Platform (release 26.09), ChEMBL record CHEMBL4297240, CC BY-SA 3.0. The Guide to Pharmacology has no record of this medicine, so its targets rest on ChEMBL alone. Trial phases are not approvals: approved uses are shown from the regulators' own records where we hold them.
Patient leaflets for Onasemnogene Abeparvovec
The package leaflet is the official guide for patients that comes with every medicine. These links go to the regulators that publish it.
- EU
- UKUnited Kingdom: Search MHRA ProductsPatient information leaflets and SmPCs for UK-licensed products.
- USUnited States: Search DailyMedFDA-approved labels, including patient and medication guides.
Leaflets differ between brands and countries; always read the one that comes with your own medicine. This is regulatory information, not medical advice.
What Zolgensma is used for
ZOLGENSMA is an adeno-associated virus (AAV) vector-based 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. Limitations of Use The safety and effectiveness of repeat administration of ZOLGENSMA have not been evaluated [see Adverse Reactions ( 6.2 )] . The use of ZOLGENSMA in patients with advanced SMA (e.g., complete paralysis of limbs, permanent ventilator-dependence) has not been evaluated [see Clinical Studies ( 14 )] . ZOLGENSMA is an adeno-associated virus (AAV) vector-based 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. ( 1 ) Limitations of Use The safety and effectiveness of repeat administratio…
How it works
12.1 Mechanism of Action ZOLGENSMA is a recombinant AAV9-based gene therapy designed to deliver a copy of the gene encoding the human SMN protein. SMA is caused by a bi-allelic mutation in the SMN1 gene, which results in insufficient SMN protein expression. Intravenous administration of ZOLGENSMA that results in cell transduction and expression of the SMN protein has been observed in two human case studies [see Clinical Pharmacology ( 12.3 )] .
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How this page is built
The facts on this page are pulled directly from official U.S. FDA datasets — they are not written from memory. Each field below names the dataset it came from, so you can check it yourself.
- FDA Drug Label (openFDA) ↗ Indications, mechanism of action and pharmacologic class
- Drugs@FDA ↗ First FDA approval date and the company holding the application
- FDA Drug Shortage Database ↗ Current US supply / shortage status
Plain-English summaries and drug-class explainers are written and reviewed by Sreepriya Prasannan (MSc Digital Transformation of Life Sciences (Innopharma Education / Griffith College); MSc & BSc Botany). Data is retrieved automatically from the sources above and cross-checked with AI-assisted verification (Anthropic's Claude) — brand and generic names are matched against the exact FDA product record so that a combination product or a different formulation cannot be mistaken for the drug on this page. An editor reviews the result before publication. We describe this in full in our editorial standards and corrections policy. The FDA data on this page was last retrieved on 11 Oct 2026. How every register is built: methodology · fixes we have made: corrections log.