Evrysdi vs Zolgensma
A daily oral treatment against a single-dose gene therapy in spinal muscular atrophy.
At a glance
Key differences
- Different classes. Evrysdi is an SMN2 splicing modifier; Zolgensma is a gene therapy. They act on different targets in the body.
- Who markets them. Evrysdi is marketed by Genentech (Roche); Zolgensma by Novartis.
What each one treats
An oral liquid medication used daily to treat spinal muscular atrophy.
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 each one works
An oral liquid medication used daily to treat spinal muscular atrophy.
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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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 6 Oct 2026. How every register is built: methodology · fixes we have made: corrections log.