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ORIGINAL RESEARCH article

Front. Pharmacol.
Sec. Drugs Outcomes Research and Policies
Volume 15 - 2024 | doi: 10.3389/fphar.2024.1486597

Safety assessment of sapropterin dihydrochloride: Real-world adverse event analysis based on the FDA Adverse Event Reporting System (FAERS)

Provisionally accepted
Jiahong Zhong Jiahong Zhong 1Xihui Yu Xihui Yu 2*Zhuomiao Lin Zhuomiao Lin 1
  • 1 Department of Clinical Pharmacy, Meizhou People's Hospital, Meizhou, Guangdong Province, China
  • 2 Department of Pharmacy, Second Affiliated Hospital of Shantou University Medical College, Shantou, China

The final, formatted version of the article will be published soon.

    Objective: Sapropterin dihydrochloride is the first drug for the therapy of phenylketonuria, which is a rare disease that occurs one of 10,000-15,000 newborns. As a result, detailed and comprehensive reports on the safety of sapropterin in large, real-world populations are required. The purpose of this study is to undertake a complete analysis of sapropterin's adverse events (AEs) using the FDA Adverse Event Reporting System (FAERS) database.Methods: We retrieved reports of adverse events with sapropterin as the principal suspect from FAERS between the first quarter of 2008 and the first quarter of 2024. The Reporting Odds Ratio (ROR), Proportional Reporting Ratio (PRR), and Bayesian Confidence Propagation Neural Network (BCPNN) were utilized to detect AE signals.Results: The study collected 4953 suspected AE cases from the FAERS database, with sapropterin as the major suspect. A total of 130 positive signals were obtained utilizing the ROR, PRP, and BCPNN. The FAERS database revealed that common clinical AEs of sapropterin included vomiting, upper respiratory infection, rhinorrhea, and a reduction in amino acid concentrations. Furthermore, we detected probable unexpected adverse events (AEs) using disproportionality analysis, including gastroesophageal reflux disease, flatulence, influenza, ear infection, viral infection, pharyngitis streptococcal, spontaneous abortion, and nephrolithiasis. Conclusion: By analyzing huge amounts of real-world data from the FAERS database, we found potential novel AEs of sapropterin using disproportionate analysis. It is advantageous for health care professionals and pharmacists to focus on efficiently managing sapropterin's high-risk adverse events, improving drug levels in clinical settings, and ensuring patient medication safety.

    Keywords: sapropterin, FAERS, adverse events, phenylketonuria, Hyperphenylalaninemia

    Received: 26 Aug 2024; Accepted: 17 Oct 2024.

    Copyright: © 2024 Zhong, Yu and Lin. 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, Department of Pharmacy, Second Affiliated Hospital of Shantou University Medical College, Shantou, China

    Disclaimer: All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.