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Volume 17
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10.3390/ph17030394
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Article

Real-World Safety Data of the Orphan Drug Onasemnogene Abeparvovec (Zolgensma®) for the SMA Rare Disease: A Pharmacovigilance Study Based on the EMA Adverse Event Reporting System

by
Rosanna Ruggiero
1,2,*,
Nunzia Balzano
1,2,
Maria Maddalena Nicoletti
3,
Gabriella di Mauro
1,2,4,
Federica Fraenza
1,2,
Maria Rosaria Campitiello
5,
Francesco Rossi
1,2,6 and
Annalisa Capuano
1,2
1
Department of Experimental Medicine, University of Campania “Luigi Vanvitelli”, 80138 Naples, Italy
2
Pharmacovigilance and Pharmacoepidemiology Regional Center of Campania Region, 80138 Naples, Italy
3
Department of Precision Medicine, University of Campania “Luigi Vanvitelli”, 80138 Naples, Italy
4
UOC Pharmacy, AORN Santobono-Pausilipon Children’s Hospital, 80122 Naples, Italy
5
Department of Obstetrics and Gynaecology and Physiopathology of Human Reproduction, ASL Salerno, 84124 Salerno, Italy
6
Department of Life Sciences, Health and Health Professions, Link Campus University, 00165 Rome, Italy
*
Author to whom correspondence should be addressed.
Pharmaceuticals 2024, 17(3), 394; https://doi.org/10.3390/ph17030394
Submission received: 26 February 2024 / Revised: 13 March 2024 / Accepted: 15 March 2024 / Published: 19 March 2024
(This article belongs to the Section Pharmacology)
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Abstract

The recent introduction of the innovative therapy, onasemnogene abeparvovec (Zolgensma®), has revolutionized the spinal muscular atrophy (SMA) therapeutic landscape. Although Zolgensma® 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® 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®, 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® 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.
Keywords: gene therapy; orphan drug; safety data; pharmacovigilance; spinal muscular atrophy gene therapy; orphan drug; safety data; pharmacovigilance; spinal muscular atrophy

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MDPI and ACS Style

Ruggiero, R.; Balzano, N.; Nicoletti, M.M.; di Mauro, G.; Fraenza, F.; Campitiello, M.R.; Rossi, F.; Capuano, A. Real-World Safety Data of the Orphan Drug Onasemnogene Abeparvovec (Zolgensma®) for the SMA Rare Disease: A Pharmacovigilance Study Based on the EMA Adverse Event Reporting System. Pharmaceuticals 2024, 17, 394. https://doi.org/10.3390/ph17030394

AMA Style

Ruggiero R, Balzano N, Nicoletti MM, di Mauro G, Fraenza F, Campitiello MR, Rossi F, Capuano A. Real-World Safety Data of the Orphan Drug Onasemnogene Abeparvovec (Zolgensma®) for the SMA Rare Disease: A Pharmacovigilance Study Based on the EMA Adverse Event Reporting System. Pharmaceuticals. 2024; 17(3):394. https://doi.org/10.3390/ph17030394

Chicago/Turabian Style

Ruggiero, Rosanna, Nunzia Balzano, Maria Maddalena Nicoletti, Gabriella di Mauro, Federica Fraenza, Maria Rosaria Campitiello, Francesco Rossi, and Annalisa Capuano. 2024. "Real-World Safety Data of the Orphan Drug Onasemnogene Abeparvovec (Zolgensma®) for the SMA Rare Disease: A Pharmacovigilance Study Based on the EMA Adverse Event Reporting System" Pharmaceuticals 17, no. 3: 394. https://doi.org/10.3390/ph17030394

APA Style

Ruggiero, R., Balzano, N., Nicoletti, M. M., di Mauro, G., Fraenza, F., Campitiello, M. R., Rossi, F., & Capuano, A. (2024). Real-World Safety Data of the Orphan Drug Onasemnogene Abeparvovec (Zolgensma®) for the SMA Rare Disease: A Pharmacovigilance Study Based on the EMA Adverse Event Reporting System. Pharmaceuticals, 17(3), 394. https://doi.org/10.3390/ph17030394

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