ORIGINAL RESEARCH article
Front. Pharmacol.
Sec. Experimental Pharmacology and Drug Discovery
Volume 16 - 2025 | doi: 10.3389/fphar.2025.1623921
This article is part of the Research TopicStrategic Drug Development for Rare and Underexplored DiseasesView all articles
Safety assessment of laronidase real-world adverse event analysis based on the FDA adverse event reporting system (FAERS)
Provisionally accepted
- 1Meizhou People's Hospital, Meizhou, China
- 2The Second Affiliated Hospital of Shantou University Medical College, Shantou, China
- 3Shantou University & the Chinese University of Hong Kong, Shantou, China
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Objective: Laronidase is the first drug of enzyme replacement therapy approved for the treatment of mucopolysaccharidosis type I (MPS I). However, its adverse events (AEs) have not been investigated in real - world settings. The aim of this study was to investigate AEs associated with laronidase using the Food and Drug Administration Adverse Event Reporting System (FAERS). Methods: Data for laronidase were acquired from the FAERS database covering Q1 2004 through Q4 2024. The Reporting Odds Ratio (ROR), Proportional Reporting Ratio (PRR), Bayesian Confidence Propagation Neural Network (BCPNN) and Multi-item Gamma Poisson Shrinker (MGPS) were employed to identify potential safety signals. Results: A total of 3,677 adverse event reports associated with laronidase were identified in the FAERS from 2004 to 2024. The results revealed that common AEs of laronidase such as pyrexia(n=465, ROR=6.23[5.68-6.83]), pneumonia(n=223, ROR=3.22[2.82-3.68]), cough(n=167, ROR=2.78[2.38-3.23]), influenza(n=114, ROR=4.95[4.12-5.95]), urticaria(n=106, ROR=2.99[2.47-3.62]), disease progression(n=101, ROR=3.95[3.25-4.81]). Furthermore, we detected probable unexpected AEs like seizures(n=75, ROR=3.1[2.47-3.89]), hydrocephalus(n=60, ROR=50.47[39.1-65.14]), blindness(n=44, ROR=5.02[3.73-6.75]), glaucoma(n=32, ROR=7.56 [5.34-10.69]). Laronidase -induced adverse reactions involved 27 System Organ Class (SOC). No significant difference in AEs was observed between sexes for laronidase. Most AEs (n=763) emerged more than 360 days following laronidase treatment. Conclusion: Our study has identified AEs associated with laronidase that could provide support for clinical monitoring and risk identification of laronidase.
Keywords: Laronidase, FAERS, adverse events, Mucopolysaccharidosis type I, Pharmacovigilance
Received: 06 May 2025; Accepted: 25 Jul 2025.
Copyright: © 2025 Lin, xue, Yang, Yu and Zhong. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence:
Xihui Yu, The Second Affiliated Hospital of Shantou University Medical College, Shantou, China
Jiahong Zhong, Meizhou People's Hospital, Meizhou, China
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