{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,26]],"date-time":"2026-09-26T09:44:38Z","timestamp":1790415878262,"version":"4.1.0"},"reference-count":48,"publisher":"MDPI AG","issue":"3","license":[{"start":{"date-parts":[[2024,3,19]],"date-time":"2024-03-19T00:00:00Z","timestamp":1710806400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Pharmaceuticals"],"abstract":"<jats:p>The recent introduction of the innovative therapy, onasemnogene abeparvovec (Zolgensma\u00ae), has revolutionized the spinal muscular atrophy (SMA) therapeutic landscape. Although Zolgensma\u00ae therapy has proven to lead to functional improvements in SMA children, some gaps in its safety profile still need to be investigated. To better characterize the Zolgensma\u00ae safety profile, we conducted a retrospective observational study, analyzing all the Individual Case Safety Reports (ICSRs) referred to it and collected in the European pharmacovigilance database between 1 January 2019 and 22 September 2023. We found 661 ICSRs related to Zolgensma\u00ae, with a growing trend in the annual reporting. The majority of the reports were sent by healthcare professionals and referred to infant females. In more than 90% of the cases, Zolgensma\u00ae was the only reported suspected drug. Out of a total of 2744 reported ADRs, increased hepatic enzymes, pyrexia, vomiting, and thrombocytopenia were the most commonly reported adverse reactions. Of these adverse reactions (ADRs), 56.9% were serious, causing or prolonging the patient\u2019s hospitalization. A total of 39 ICSRs related to cases with a fatal outcome. Alterations in the heart rhythm, acute hepatic failure, and hepatic cytolysis emerged among the cardiac and hepatic disorders, respectively.<\/jats:p>","DOI":"10.3390\/ph17030394","type":"journal-article","created":{"date-parts":[[2024,3,19]],"date-time":"2024-03-19T09:39:31Z","timestamp":1710841171000},"page":"394","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":23,"title":["Real-World Safety Data of the Orphan Drug Onasemnogene Abeparvovec (Zolgensma\u00ae) for the SMA Rare Disease: A Pharmacovigilance Study Based on the EMA Adverse Event Reporting System"],"prefix":"10.3390","volume":"17","author":[{"given":"Rosanna","family":"Ruggiero","sequence":"first","affiliation":[{"name":"Department of Experimental Medicine, University of Campania \u201cLuigi Vanvitelli\u201d, 80138 Naples, Italy"},{"name":"Pharmacovigilance and Pharmacoepidemiology Regional Center of Campania Region, 80138 Naples, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nunzia","family":"Balzano","sequence":"additional","affiliation":[{"name":"Department of Experimental Medicine, University of Campania \u201cLuigi Vanvitelli\u201d, 80138 Naples, Italy"},{"name":"Pharmacovigilance and Pharmacoepidemiology Regional Center of Campania Region, 80138 Naples, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maria Maddalena","family":"Nicoletti","sequence":"additional","affiliation":[{"name":"Department of Precision Medicine, University of Campania \u201cLuigi Vanvitelli\u201d, 80138 Naples, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gabriella","family":"di Mauro","sequence":"additional","affiliation":[{"name":"Department of Experimental Medicine, University of Campania \u201cLuigi Vanvitelli\u201d, 80138 Naples, Italy"},{"name":"Pharmacovigilance and Pharmacoepidemiology Regional Center of Campania Region, 80138 Naples, Italy"},{"name":"UOC Pharmacy, AORN Santobono-Pausilipon Children\u2019s Hospital, 80122 Naples, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Federica","family":"Fraenza","sequence":"additional","affiliation":[{"name":"Department of Experimental Medicine, University of Campania \u201cLuigi Vanvitelli\u201d, 80138 Naples, Italy"},{"name":"Pharmacovigilance and Pharmacoepidemiology Regional Center of Campania Region, 80138 Naples, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maria Rosaria","family":"Campitiello","sequence":"additional","affiliation":[{"name":"Department of Obstetrics and Gynaecology and Physiopathology of Human Reproduction, ASL Salerno, 84124 Salerno, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francesco","family":"Rossi","sequence":"additional","affiliation":[{"name":"Department of Experimental Medicine, University of Campania \u201cLuigi Vanvitelli\u201d, 80138 Naples, Italy"},{"name":"Pharmacovigilance and Pharmacoepidemiology Regional Center of Campania Region, 80138 Naples, Italy"},{"name":"Department of Life Sciences, Health and Health Professions, Link Campus University, 00165 Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Annalisa","family":"Capuano","sequence":"additional","affiliation":[{"name":"Department of Experimental Medicine, University of Campania \u201cLuigi Vanvitelli\u201d, 80138 Naples, Italy"},{"name":"Pharmacovigilance and Pharmacoepidemiology Regional Center of Campania Region, 80138 Naples, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2024,3,19]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"3877","DOI":"10.1007\/s00018-018-2849-1","article-title":"The Role of Survival Motor Neuron Protein (SMN) in Protein Homeostasis","volume":"75","author":"Chaytow","year":"2018","journal-title":"Cell. 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P\u00e9diatrie"},{"key":"ref_9","doi-asserted-by":"crossref","unstructured":"Crisafulli, S., Boccanegra, B., Vitturi, G., Trifir\u00f2, G., and De Luca, A. (2023). Pharmacological Therapies of Spinal Muscular Atrophy: A Narrative Review of Preclinical, Clinical\u2013Experimental, and Real-World Evidence. Brain Sci., 13.","DOI":"10.3390\/brainsci13101446"},{"key":"ref_10","first-page":"100822","article-title":"Gene Replacement Therapy in Spinal Muscular Atrophy: Filling the Data Gaps","volume":"37","author":"Hagenacker","year":"2024","journal-title":"Lancet Reg. Health\u2014Eur."},{"key":"ref_11","doi-asserted-by":"crossref","first-page":"1713","DOI":"10.1056\/NEJMoa1706198","article-title":"Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy","volume":"377","author":"Mendell","year":"2017","journal-title":"N. Engl. J. 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