Real-World Safety Data of the Orphan Drug Onasemnogene Abeparvovec (Zolgensma<sup>®</sup>) for the SMA Rare Disease: A Pharmacovigilance Study Based on the EMA Adverse Event Reporting System
The recent introduction of the innovative therapy, onasemnogene abeparvovec (Zolgensma<sup>®</sup>), has revolutionized the spinal muscular atrophy (SMA) therapeutic landscape. Although Zolgensma<sup>®</sup> therapy has proven to lead to functional improvements in SMA childre...
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MDPI AG
2024-03-01
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| Online Access: | https://www.mdpi.com/1424-8247/17/3/394 |
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| author | Rosanna Ruggiero Nunzia Balzano Maria Maddalena Nicoletti Gabriella di Mauro Federica Fraenza Maria Rosaria Campitiello Francesco Rossi Annalisa Capuano |
| author_facet | Rosanna Ruggiero Nunzia Balzano Maria Maddalena Nicoletti Gabriella di Mauro Federica Fraenza Maria Rosaria Campitiello Francesco Rossi Annalisa Capuano |
| author_sort | Rosanna Ruggiero |
| collection | DOAJ |
| description | The recent introduction of the innovative therapy, onasemnogene abeparvovec (Zolgensma<sup>®</sup>), has revolutionized the spinal muscular atrophy (SMA) therapeutic landscape. Although Zolgensma<sup>®</sup> 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<sup>®</sup> 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<sup>®</sup>, 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<sup>®</sup> 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’s 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. |
| first_indexed | 2024-04-24T17:54:44Z |
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| institution | Directory Open Access Journal |
| issn | 1424-8247 |
| language | English |
| last_indexed | 2024-04-24T17:54:44Z |
| publishDate | 2024-03-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Pharmaceuticals |
| spelling | doaj.art-1ecf90f4db12436bbb1cb33b32f64c232024-03-27T13:59:29ZengMDPI AGPharmaceuticals1424-82472024-03-0117339410.3390/ph17030394Real-World Safety Data of the Orphan Drug Onasemnogene Abeparvovec (Zolgensma<sup>®</sup>) for the SMA Rare Disease: A Pharmacovigilance Study Based on the EMA Adverse Event Reporting SystemRosanna Ruggiero0Nunzia Balzano1Maria Maddalena Nicoletti2Gabriella di Mauro3Federica Fraenza4Maria Rosaria Campitiello5Francesco Rossi6Annalisa Capuano7Department of Experimental Medicine, University of Campania “Luigi Vanvitelli”, 80138 Naples, ItalyDepartment of Experimental Medicine, University of Campania “Luigi Vanvitelli”, 80138 Naples, ItalyDepartment of Precision Medicine, University of Campania “Luigi Vanvitelli”, 80138 Naples, ItalyDepartment of Experimental Medicine, University of Campania “Luigi Vanvitelli”, 80138 Naples, ItalyDepartment of Experimental Medicine, University of Campania “Luigi Vanvitelli”, 80138 Naples, ItalyDepartment of Obstetrics and Gynaecology and Physiopathology of Human Reproduction, ASL Salerno, 84124 Salerno, ItalyDepartment of Experimental Medicine, University of Campania “Luigi Vanvitelli”, 80138 Naples, ItalyDepartment of Experimental Medicine, University of Campania “Luigi Vanvitelli”, 80138 Naples, ItalyThe recent introduction of the innovative therapy, onasemnogene abeparvovec (Zolgensma<sup>®</sup>), has revolutionized the spinal muscular atrophy (SMA) therapeutic landscape. Although Zolgensma<sup>®</sup> 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<sup>®</sup> 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<sup>®</sup>, 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<sup>®</sup> 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’s 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.https://www.mdpi.com/1424-8247/17/3/394gene therapyorphan drugsafety datapharmacovigilancespinal muscular atrophy |
| spellingShingle | Rosanna Ruggiero Nunzia Balzano Maria Maddalena Nicoletti Gabriella di Mauro Federica Fraenza Maria Rosaria Campitiello Francesco Rossi Annalisa Capuano Real-World Safety Data of the Orphan Drug Onasemnogene Abeparvovec (Zolgensma<sup>®</sup>) for the SMA Rare Disease: A Pharmacovigilance Study Based on the EMA Adverse Event Reporting System Pharmaceuticals gene therapy orphan drug safety data pharmacovigilance spinal muscular atrophy |
| title | Real-World Safety Data of the Orphan Drug Onasemnogene Abeparvovec (Zolgensma<sup>®</sup>) for the SMA Rare Disease: A Pharmacovigilance Study Based on the EMA Adverse Event Reporting System |
| title_full | Real-World Safety Data of the Orphan Drug Onasemnogene Abeparvovec (Zolgensma<sup>®</sup>) for the SMA Rare Disease: A Pharmacovigilance Study Based on the EMA Adverse Event Reporting System |
| title_fullStr | Real-World Safety Data of the Orphan Drug Onasemnogene Abeparvovec (Zolgensma<sup>®</sup>) for the SMA Rare Disease: A Pharmacovigilance Study Based on the EMA Adverse Event Reporting System |
| title_full_unstemmed | Real-World Safety Data of the Orphan Drug Onasemnogene Abeparvovec (Zolgensma<sup>®</sup>) for the SMA Rare Disease: A Pharmacovigilance Study Based on the EMA Adverse Event Reporting System |
| title_short | Real-World Safety Data of the Orphan Drug Onasemnogene Abeparvovec (Zolgensma<sup>®</sup>) for the SMA Rare Disease: A Pharmacovigilance Study Based on the EMA Adverse Event Reporting System |
| title_sort | real world safety data of the orphan drug onasemnogene abeparvovec zolgensma sup r sup for the sma rare disease a pharmacovigilance study based on the ema adverse event reporting system |
| topic | gene therapy orphan drug safety data pharmacovigilance spinal muscular atrophy |
| url | https://www.mdpi.com/1424-8247/17/3/394 |
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