Adverse Event Profile Differences Between Eliglustat and Miglustat: A Pharmacovigilance Study using the U.S. Food and Drug Administration Adverse Event Reporting System

Abstract

Aims

Both eliglustat and miglustat are substrate reduction therapies targeting glucosylceramide synthase; yet, their safety profile has not been comprehensively analyzed. This study analyzes adverse events associated with both drugs using the U.S. Food and Drug Administration Adverse Event Reporting System to provide insights for clinical safety.

Methods

Adverse events were classified by MedDRA System Organ Class (SOC, v26.1). Adverse event signals were mined by disproportionality analyses, including the reporting odds ratio, the proportional reporting ratio, the multi-item gamma Poisson shrinker algorithms, and the Bayesian confidence propagation neural network.

Results

A total of 1,223 and 980 adverse event reports were retrieved from eliglustat and miglustat, respectively, involving 27 System Organ Class categories each. Some positive signals were consistent with the drug labels, including dyspepsia identified in eliglustat and diarrhoea identified in miglustat. We also identified unexpected signals not listed on the drug labels, such as paresthesia, dry skin, and ichthyosis for eliglustat and dysphagia for miglustat. For patients treated with eliglustat and miglustat, the majority of adverse events manifested more than 1 year after the initiation of therapy. Notably, male patients treated with eliglustat have the significantly higher incidence of weight increase and dry skin. Female patients treated with miglustat have the significantly higher incidence of dysphagia and cognitive disorder.

Conclusions

In the clinical administration of eliglustat and miglustat, clinicians need to monitor the effects of adverse events varied by gender and to pay more attention to new adverse event signals.

Keywords

Miglustat - adverse events - gaucher’s disease - disproportionality analysis

Publication History

Received: 22 August 2025

Accepted after revision: 16 January 2026

Article published online:
05 February 2026

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