Cited by 337Neurogenetic and Muscular Disorders ResearchVirus-based gene therapy researchLysosomal Storage Disorders Research

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Onasemnogene Abeparvovec: First Global Approval

Hoy, Sheridan M.·2019·Drugs

DOI 10.1007/s40265-019-01162-5

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Abstract

Onasemnogene abeparvovec (onasemnogene abeparvovec-xioi; formerly AVXS-101; ZOLGENSMA ® ) is an adeno-associated viral vector-based gene therapy designed to deliver a functional copy of the human survival motor neuron (SMN) gene to the motor neuron cells of patients with spinal muscular atrophy (SMA). It has been developed by AveXis, a Novartis company, and was approved in May 2019 in the USA for the treatment of paediatric patients aged 14 vector genomes per kg of bodyweight, administered as a single intravenous infusion over 60 min. Regulatory assessments for this formulation of onasemnogene abeparvovec are underway in the EU and Japan; an intrathecal formulation is currently undergoing clinical development in the USA. This article summarizes the milestones in the development of onasemnogene abeparvovec leading to this first approval for the treatment of paediatric patients aged < 2 years with SMA and bi-allelic mutations in SMN1.

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1 retracted

RETRACTED ARTICLE: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN
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Retracted
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
Mendell, J. R., Al-Zaidy, S., Shell, R., Arnold, W. D., Rodino-Klapac, L. R., Prior, T. W., Lowes, L., Alfano, L., Berry, K., Church, K., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1706198 · Cited by 2,381
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Prevalence, incidence and carrier frequency of 5q-linked spinal muscular atrophy - a literature review
Verhaart, I. E. C., Robertson, A., Wilson, I. J., Aartsma-Rus, A., Cameron, S., Jones, C. C., Cook, S. F., & Lochmüller, H. (2017) · Orphanet Journal of Rare Diseases · doi:10.1186/s13023-017-0671-8 · Cited by 739

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Background
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Background
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Nanotechnology and CRISPR/Cas-Mediated Gene Therapy Strategies: Potential Role for Treating Genetic Disorders
Kaur, G., Arora, J., Sodhi, A. S., Bhatia, S., & Batra, N. (2024) · Molecular Biotechnology · doi:10.1007/s12033-024-01301-8 · Cited by 14
Adeno-Associated Virus Delivery Limitations for Neurological Indications
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Potential Applications for Targeted Gene Therapy to Protect Against Anthracycline Cardiotoxicity
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Boosting transfection efficiency: A systematic study using layer-by-layer based gene delivery platform.
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Investigating Immune Responses to the scAAV9-HEXM Gene Therapy Treatment in Tay–Sachs Disease and Sandhoff Disease Mouse Models
Kot, S., Karumuthil‐Melethil, S., Woodley, E., Zaric, V., Thompson, P., Chen, Z., Lykken, E. A., Keimel, J. G., Kaemmerer, W. F., Gray, S. J., et al. (2021) · International Journal of Molecular Sciences · doi:10.3390/ijms22136751 · Cited by 13
“Food and Drug Administration (Zolgensma® FDA [29]).”
Gene therapy using Aβ variants for amyloid reduction
Park, K., Wood, C. A., Li, J., Taylor, B. C., Oh, S.-W., Young, N. L., & Jankowsky, J. L. (2021) · Molecular Therapy · doi:10.1016/j.ymthe.2021.02.026 · Cited by 11
Limited potential of AAV-mediated gene therapy in transducing human mesenchymal stem cells for bone repair applications
Bougioukli, S., Chateau, M., Morales, H., Vakhshori, V., Sugiyama, O., Oakes, D. A., Longjohn, D. B., Cannon, P. M., & Lieberman, J. R. (2020) · Gene Therapy · doi:10.1038/s41434-020-0182-4 · Cited by 12
Evaluating Perceived Fatigue within an Adult Spinal Muscular Atrophy Population
Belter, L. T., Peterson, I. S., & Jarecki, J. (2023) · Neurology and Therapy · doi:10.1007/s40120-023-00552-y · Cited by 11
Vector Affinity and Receptor Distribution Define Tissue-Specific Targeting in an Engineered AAV Capsid
Martino, R. A., Wang, Q., Xu, H. Y., Hu, G. B., Bell, P. L., Arroyo, E. J., Sims, J. J., & Wilson, J. M. (2023) · Journal of Virology · doi:10.1128/jvi.00174-23 · Cited by 13
“Food and Drug Administration granted final approval for Zolgensma, a genetic therapy for pediatric patients suffering from spinal muscular atrophy (1, 2).”
Background
Diagnostic experiences of Duchenne families and their preferences for newborn screening: A mixed‐methods study
Crossnohere, N., Armstrong, N., Fischer, R., & Bridges, J. F. P. (2022) · American Journal of Medical Genetics. Part C, Seminars in Medical Genetics · doi:10.1002/ajmg.c.31992 · Cited by 10
“Gene therapies are routinely administered in infancy for spinal muscular atrophy, (Hoy, 2019) and there is emerging data to suggest that administration in infancy might be similarly effective in muscular dystrophy.”
Background
Unlocking the potential: advancements and applications of gene therapy in severe disorders
Ingle, R. G., Elossaily, G. M., Ansari, M. N., & Makhijani, S. (2025) · Annals medicus · doi:10.1080/07853890.2025.2516697 · Cited by 9
Improving AAV Production Yield and Quality for Different Serotypes Using Distinct Processing Methods
Khaparde, A., Patra, R., Roy, S., R, S. B. G., & Ghosh, A. (2025) · ACS Omega · doi:10.1021/acsomega.4c10900 · Cited by 9
“22 Hemgenix and Roctavian are among latest AAV5-based gene therapies for Hemophilia B and Hemophilia A respectively.”
From roadmap to a sustainable end-to-end individualized therapy pathway
Jonker, A., Tataru, E., Dimmock, D., Bateman-House, A., Graessner, H., Baynam, G., Augustine, E., Jaffe, A., Pasmooij, A. M., Iliach, O., et al. (2025) · Therapeutic Advances in Rare Disease · doi:10.1177/26330040251339204 · Cited by 9
Fatal outcomes following onasemnogene abeparvovec in advanced-stage spinal muscular atrophy
Pongsakornkullachart, P., Kulsirichawaroj, P., Kongkasuwan, R., Tovichien, P., Jitwongwai, S., Kanjanauthai, S., Preeprem, N., Limpaninlachat, S., Sermpon, N., & Sanmaneechai, O. (2025) · Gene Therapy · doi:10.1038/s41434-025-00535-8 · Cited by 9
“OA is indicated for the treatment of SMA patients under 2 years of age with biallelic mutations in the SMN1 gene [10] or body weight less than 21 kg, independent age [11].”
Targeting STMN2 for neuroprotection and neuromuscular recovery in Spinal Muscular Atrophy: evidence from in vitro and in vivo SMA models
Pagliari, E., Taiana, M. M., Manzini, P., Sali, L., Quetti, L., Bertolasi, L., Oldoni, S., Melzi, V., Comi, G. P., Corti, S. P., et al. (2024) · Cellular and Molecular Life Sciences · doi:10.1007/s00018-024-05550-3 · Cited by 11
“…cause of infant mortality, characterized by the degeneration of spinal motor neurons (MNs) inclusion of exon 7 in messenger RNA (mRNA), as well as non-replicating adeno-associated virus (AAV)-based gene therapy, delivering a functional SMN1 cDNA efficiently to affected MNs [ 16].”
Adeno-associated virus vector intraperitoneal injection induces colonic mucosa and submucosa transduction and alters the diversity and composition of the faecal microbiota in rats
Ma, L., Lian, J.-X., Bai, Y., Shang, M.-J., Zhang, Z., Wu, F., Chen, J., Meng, X.-B., Zheng, J., Li, T. X., et al. (2022) · Frontiers in Cellular and Infection Microbiology · doi:10.3389/fcimb.2022.1028380 · Cited by 10
“…FDA for the treatment of the early childhood blindness disease Leber congenital amaurosis (Kumaran et al., 2018) and spinal muscular atrophy (SMA) (Hoy, 2019), and almost all gene editing work applied in the clinic relies on AAV vectors to deliver a genetic payload (Dunbar et al., 2018; High and…”
BackgroundInfluential
Regenerative Medicine and Angiogenesis; Focused on Cardiovascular Disease
Moradi, S. Z., Jalili, F., Hoseinkhani, Z., & Mansouri, K. (2021) · Advanced Pharmaceutical Bulletin · doi:10.34172/apb.2022.072 · Cited by 9
Safety Considerations for Nanoparticle Gene Delivery in Pediatric Brain Tumors
Luly, K. M., Choi, J. H., Rui, Y., Green, J. J., & Jackson, E. M. (2020) · Nanomedicine · doi:10.2217/nnm-2020-0110 · Cited by 13
Multi-omics profiling in spinal muscular atrophy (SMA): investigating lipid and metabolic alterations through longitudinal CSF analysis of Nusinersen-treated patients
Zandl-Lang, M., Züllig, T., Holzer, M., Eichmann, T. O., Darnhofer, B., Schwerin-Nagel, A., Zobel, J., Haidl, H., Biebl, A., Köfeler, H., et al. (2025) · Journal of Neurology · doi:10.1007/s00415-025-12909-4 · Cited by 8
“…onasemnogene abeparvovec, a gene therapy applied by a one-time intravenous infusion of an adeno-associated virus vector for SMN1 correction has been approved by the FDA for patients with SMA below 2 years of age with bial-lelic mutations of the SMN1 gene and up to three copies of the SMN2 gene [9].”
Is Duchenne gene therapy a suitable treatment despite its immunogenic class effect?
Tang, A., & Yokota, T. (2024) · Expert Opinion on Drug Safety · doi:10.1080/14740338.2024.2447072 · Cited by 8
Characterization of SMA type II skeletal muscle from treated patients shows OXPHOS deficiency and denervation
Grandi, F. C., Astord, S., Gidaja, E., Pezet, S., Mazzucchi, S., Chapart, M., Vasseur, S., Mamchaoui, K., & Smeriglio, P. (2024) · JCI Insight · doi:10.1172/jci.insight.180992 · Cited by 12
“One main challenge for type II SMA patients treated with these drugs is ongoing muscle fatigue, limited mobility, and other skeletal problems.”
Gene replacement therapy for spinal muscular atrophy: safety and preliminary efficacy in a Brazilian cohort
Mendonça, R. D. H., Ortega, A., Matsui, C., van der Linden, V., Kerstenetzky, M., Grossklauss, L. F., Silveira, E. L., Polido, G. J., & Zanoteli, E. (2024) · Gene Therapy · doi:10.1038/s41434-024-00456-y · Cited by 12
Targeted gene delivery systems for T-cell engineering
Wang, F., Huang, Y., Li, J., Zhou, W., & Wang, W. (2024) · Cellular Oncology · doi:10.1007/s13402-024-00954-6 · Cited by 11
Gene therapy for selected neuromuscular and trinucleotide repeat disorders – An insight to subsume South Asia for multicenter clinical trials
Wijekoon, N., Gonawala, L., Ratnayake, P., Sirisena, D., Gunasekara, H., Dissanayake, A., Senanayake, S., Keshavaraj, A., Hathout, Y., Steinbusch, H., et al. (2023) · IBRO Neuroscience Reports · doi:10.1016/j.ibneur.2023.01.009 · Cited by 8
The 46.1 Antibody Mediates Neurotensin Uptake into the CNS and the Effects Depend on the Route of Intravenous Administration
Georgieva, J. V., Katt, M. E., Zhou, Y., Umlauf, B. J., Wenthur, C. J., & Shusta, E. V. (2022) · Pharmaceutics · doi:10.3390/pharmaceutics14081706 · Cited by 11
“However, an AAV vector with a similar administration route-dependent CNS exposure [21] is already in clinical use [52], and warrants the continued discussion of how the administration route could impact the CNS delivery, particularly with a targeting motif like scFv 46.”
Background
Case Report: Whole-Exome Sequencing With MLPA Revealed Variants in Two Genes in a Patient With Combined Manifestations of Spinal Muscular Atrophy and Duchenne Muscular Dystrophy
Xia, Y., Feng, Y., Xu, L., Chen, X.-Y., Gao, F., & Mao, S. (2021) · Frontiers in Genetics · doi:10.3389/fgene.2021.605611 · Cited by 8
“Nevertheless, Zolgensma is currently indicated for the treatment of patients who have SMA type I or up to 3 copies of the SMN2 gene, and the most crucial issue is whether the safety of high-dose virus vector and systemic administration requires further evaluation (Hoy, 2019).”
Background
Engineering precision therapies: lessons and motivations from the clinic
Xie, M., Viviani, M., & Fussenegger, M. (2020) · Synthetic Biology · doi:10.1093/synbio/ysaa024 · Cited by 6
Novel Therapies for Spinal Muscular Atrophy are Likely Changing the Patient Phenotype.
Vu-Han, T.-L., Weiss, C., & Pumberger, M. (2020) · The spine journal · doi:10.1016/j.spinee.2020.08.013 · Cited by 8
Postmarketing adverse events associated with onasemnogene abeparvovec: a real-world pharmacovigilance study
Chen, T., Chen, Q., Ye, J., Wu, Y., Liu, T., & Zhang, Y. (2025) · Orphanet Journal of Rare Diseases · doi:10.1186/s13023-025-03715-2 · Cited by 7
“The most frequently reported adverse effects are elevated transaminase levels, vomiting, thrombocytopenia, and troponin I increase [20].”
Clinicogenetic characterization and response to disease-modifying therapies in spinal muscular atrophy: real-world experience from a reference center in Southern Brazil
de Albuquerque, A. L. A., Chadanowicz, J. K., Bevilacqua, I. P., Staub, A. L. P., Winckler, P., Silva, P. Z. D., Fagondes, S., Ferrari, R. S., Trojahn, C. D. D. O., Sacharuk, V. Z., et al. (2024) · Jornal de Pediatria · doi:10.1016/j.jped.2024.07.011 · Cited by 7
Update on Inherited Pediatric Motor Neuron Diseases: Clinical Features and Outcome
Trabacca, A., Ferrante, C., Oliva, M. C., Fanizza, I., Gallo, I., & Rinaldis, M. D. (2024) · Genes · doi:10.3390/genes15101346 · Cited by 8
“However, potential drawbacks include a lack of long-term efficacy and safety data [42].”
Background
An interpretable data-driven prediction model to anticipate scoliosis in spinal muscular atrophy in the era of (gene-) therapies
Vu-Han, T.-L., Schettino, R. B., Weiss, C., Perka, C., Winkler, T., Sunkara, V., & Pumberger, M. (2024) · Scientific Reports · doi:10.1038/s41598-024-62720-w · Cited by 7
Analysis of the impact of pluronic acid on the thermal stability and infectivity of AAV6.2FF
Thomas, S. P., Spinelli, M. M., Rghei, A. D., Lopes, J. A., Zielinska, N., Mcleod, B. M., Pei, Y.-L., Zhang, W., Thébaud, B., Karimi, K., et al. (2024) · BMC Biotechnology · doi:10.1186/s12896-024-00853-6 · Cited by 7
Corneal confocal microscopy reveals small nerve fibre loss correlating with motor function in adult spinal muscular atrophy
Thimm, A., Brakemeier, S., Dağ, M., Rosales, J., Stolte, B., Kleinschnitz, C., Stettner, M., & Hagenacker, T. (2023) · European Journal of Neurology · doi:10.1111/ene.15852 · Cited by 7
“Nusinersen, an antisense oligonucleotide administered intrathecally that increases the expression of SMN protein [6– 8], risdiplam, an orally administered small molecule modifying SMN2 gene splicing [9], and onasemnogene abeparvovec, a nonreplicating recombinant adenovirus- associated virus serotype 9 (AAV9) containin…”
Background
Gene regulatory and gene editing tools and their applications for retinal diseases and neuroprotection: From proof-of-concept to clinical trial
Altay, H. Y., Özdemir, F., Afghah, F., Kilinç, Z., Ahmadian, M., Tschopp, M., & Ağca, C. (2022) · Frontiers in Neuroscience · doi:10.3389/fnins.2022.924917 · Cited by 9
“Onasemnogene abeparvovec (Zolgensma) is another gene replacement therapy that delivers the functional copy of survival of motor neuron 1 (SMN1) gene using AAV9 for the treatment of spinal muscular atrophy (SMA), a devastating neuromuscular disease (Hoy, 2019; Day et al., 2021; Figure 1A).”
Background
Macrophage Depletion via Clodronate Pretreatment Reduces Transgene Expression from AAV Vectors In Vivo
Yu, D. L., Chow, N., Bridle, B. W., & Wootton, S. K. (2021) · Viruses · doi:10.3390/v13102002 · Cited by 6
“The recent regulatory approval of Luxturna, an AAV gene therapy vector designed to treat patients with a rare form of inherited vision loss [1], and Zolgensma, an AAV gene therapy for type 1 spinal muscular atrophy (SMA) [2], highlights the increasing success such gene therapy strategies are experiencing in the clinic…”
Isolating Natural Adeno-Associated Viruses from Primate Tissues with a High-Fidelity Polymerase
Wang, Q., Nambiar, K., & Wilson, J. M. (2021) · Human Gene Therapy · doi:10.1089/hum.2021.055 · Cited by 8
Muscle overexpression of Klf15 via an AAV8-Spc5-12 construct does not provide benefits in spinal muscular atrophy mice
Ahlskog, N., Hayler, D., Krueger, A., Kubinski, S., Claus, P., Hammond, S., Wood, M. J., Yáñez-Muñoz, R., & Bowerman, M. (2019) · Gene Therapy · doi:10.1038/s41434-020-0146-8 · Cited by 7
Adaptive Machine Learning Framework for Optimizing the Affinity Purification of Adeno‐Associated Viral Vectors
Idanwekhai, K., Shastry, S., Hurst, M. R., Minzoni, A., Barbieri, E., Remmler, L. A., Muratov, E. N., Daniele, M. A.-A., Menegatti, S., & Tropsha, A. (2026) · Biotechnology and Bioengineering · doi:10.1002/bit.70159 · Cited by 9
“…neuronal tissues, thus promoting its adoption in gene therapies for disorders such as spinal muscular atrophy (SMA) and muscular dystrophies, and culminating in the 2019 approval of Zolgensma as a cure for SMA (Hoy 2019; Kotulska et al. 2021; BioSpace 2019; Hatanaka et al. 2024; Gray et al. 2013).”
Application of the Insect Cell-Baculovirus Expression Vector System in Adeno-Associated Viral Production
Wang, F., Sun, J., Guo, W., & Wu, Y. (2024) · Applied Sciences · doi:10.3390/app142310948 · Cited by 6
The Relevance of Spinal Muscular Atrophy Biomarkers in the Treatment Era
Maretina, M. A., Koroleva, V., Shchugareva, L. M., Glotov, A. S., & Kiselev, A. (2024) · Biomedicines · doi:10.3390/biomedicines12112486 · Cited by 9
“Later, onasemnogene abeparvovec (Zolgensma ® , Durham, NC, USA), representing an AAV9 vector containing the SMN1 gene, and small-molecule SMN2 splicing modulator Risdiplam (Evrysdi ® , San Francisco, CA, USA) were approved for treatment of SMA as well [16,17].”
Native Digestion and Shotgun Proteomics for Host Cell Protein Profiling of Adeno-Associated Viruses.
Lodge, J., Huang, L., Lian, Z., Qian, J., & Tian, Y. (2024) · Analytical Chemistry · doi:10.1021/acs.analchem.4c00893 · Cited by 6
Onasemnogene abeparvovec gene therapy for spinal muscular atrophy: A cohort study from the United Arab Emirates
Mundada, V., Narayan, O., Arora, S., Beri, N., Abusamra, R., Mullasery, D., & Parashar, D. (2024) · Muscle and Nerve · doi:10.1002/mus.28222 · Cited by 6
ATMP Environmental Exposure Assessment in European Healthcare Settings: A Systematic Review of the Literature
Damerval, M., Fagnoni-Legat, C., Louvrier, A., Fischer, S., Limat, S., Clairet, A., Nerich, V., Madelaine, I., & Kroemer, M. (2021) · Frontiers in Medicine · doi:10.3389/fmed.2021.713047 · Cited by 9
Isolating Human Monoclonal Antibodies Against Adeno-Associated Virus From Donors With Pre-existing Immunity
Giles, A. R., Calcedo, R., Tretiakova, A. P., & Wilson, J. M. (2020) · Frontiers in Immunology · doi:10.3389/fimmu.2020.01135 · Cited by 7

Authors: Hoy, S. M.
Affiliations: Springer Nature (New Zealand) (NZ)
Corresponding author: Hoy, Sheridan M.
Venue: Drugs
Publisher: Springer Science and Business Media LLC
Published: 2019-07
Volume: 79
Issue: 11
Pages: 1255-1262
ISSN: 0012-6667 (ISSN-L)
1179-1950
DOI: 10.1007/s40265-019-01162-5
OpenAlex: W2955699452
Cited by: 337
References: 0
Citations by year: 2026: 31 · 2025: 36 · 2024: 54 · 2023: 42 · 2022: 38 · 2021: 68 · 2020: 58 · 2019: 9
FWCI: 23.71
Type: journal article
Language: en
Open access: closed
Licence: www.springer.com/tdm (tdm)
www.springer.com/tdm (vor)
Locations: Drugs (publishedVersion)
PubMed (publishedVersion)
Topics: Health Sciences › Medicine › Genetics (clinical) › Neurogenetic and Muscular Disorders Research
Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
Health Sciences › Medicine › Genetics (clinical) › Lysosomal Storage Disorders Research
Keywords: onasemnogene abeparvovec, Zolgensma, spinal muscular atrophy, gene therapy, adeno-associated virus vectors, SMN1, survival motor neuron protein, pediatric patients, regulatory approval, drug development milestones
MeSH terms: Dependovirus, Genetic Vectors, Humans, Infant, Infant, Newborn, Infusions, Intravenous, Motor Neurons, Muscular Atrophy, Spinal, Mutation, United States, United States Food and Drug Administration, Genetic Therapy, Drug Approval, SMN Complex Proteins

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Onasemnogene Abeparvovec: First Global Approval

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