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Real-world pharmacovigilance analysis of galsulfase: a study based on the FDA adverse event reporting system (FAERS)
Shangze Li1, Runcheng Huang2, Yuanyuan Meng3
1Department of Orthopedics, The Second Affiliated Hospital (Changzheng Hospital), Naval Medical University, Shanghai, China.
Background:
Associated with enzyme deficiencies causing glycosaminoglycans (GAGs) accumulation, mucopolysaccharidosis type VI (MPS VI) is lysosomal storage disorder. In the treatment of MPS VI, galsulfase (Naglazyme) is commonly used as an enzyme replacement therapy (ERT). There remains a need for comprehensive real-world data on its safety and associated adverse events (AEs).
Objective:
An analysis of the FDA Adverse Event Reporting System (FAERS) database will be conducted to identify potential risks and adverse reactions associated with galsulfase in real-life settings.
Methods:
The FAERS database was used to extract data from Q2 2005 to Q4 2023. A total of 20,281,876 reports were analyzed after duplicate elimination, with 3,195 AE reports related to galsulfase identified. The association between galsulfase and AEs was investigated by utilizing four algorithms: reporting odds ratio (ROR), proportional reporting ratio (PRR), Bayesian confidence propagation neural network (BCPNN), and multi-item gamma Poisson shrinker (MGPS). The analysis focused on the timing of onset, signs of AEs, and clinical significance.
Results:
Twenty seven organ systems were involved, and significant system organ classes (SOCs) included respiratory, thoracic and mediastinal disorders, and infections and infestations. At the PT level, 72 PTs corresponding to 15 SOCs were identified, with some AEs not previously mentioned in the product label. AEs associated with galsulfase had a median onset time of 1,471 days, with over half of the cases occurred within the first 5 years of treatment initiation.
Conclusion:
This investigation delivers an exhaustive and indicative assessment of galsulfase's safety profile, grounded in authentic, real-world evidence. The findings emphasis the importance of continuous safety surveillance and the emergence of new AEs. The identification of previously unreported urologic adverse events, such as glomerulonephritis membranous and nephritic syndrome, warrants further investigation. The study emphasizes the need for enhanced pharmacovigilance to ensure patient safety and the effectiveness of galsulfase treatment.
Insights
This study analyzed real-world data on galsulfase (Naglazyme) for mucopolysaccharidosis type VI (MPS VI), revealing new adverse events like kidney issues. Continuous safety monitoring is crucial for effective enzyme replacement therapy (ERT).
Area of Science:
- Pharmacovigilance and Drug Safety
- Lysosomal Storage Disorders
- Enzyme Replacement Therapy
Background:
- Mucopolysaccharidosis type VI (MPS VI) is a lysosomal storage disorder caused by GAGs accumulation.
- Galsulfase (Naglazyme) is a key enzyme replacement therapy (ERT) for MPS VI.
- Real-world safety data and adverse events (AEs) for galsulfase require comprehensive analysis.
Purpose of the Study:
- To analyze the FDA Adverse Event Reporting System (FAERS) database for galsulfase safety.
- To identify potential risks and adverse reactions associated with galsulfase in real-world settings.
- To assess the safety profile and identify emerging adverse events of galsulfase treatment.
Main Methods:
- Extracted data from the FAERS database (Q2 2005 - Q4 2023), analyzing 3,195 galsulfase-related AE reports.
- Employed four algorithms (ROR, PRR, BCPNN, MGPS) to investigate the association between galsulfase and AEs.
- Focused on AE onset timing, signs, and clinical significance.
Main Results:
- Identified 72 specific adverse events across 15 organ systems, including respiratory and infectious disorders.
- Observed a median AE onset time of 1,471 days, with over half occurring within the first 5 years of treatment.
- Detected previously unreported urologic adverse events, such as membranous glomerulonephritis and nephritic syndrome.
Conclusions:
- This study provides real-world evidence on the galsulfase safety profile, highlighting the emergence of new AEs.
- The identification of previously unreported urologic adverse events necessitates further investigation.
- Enhanced pharmacovigilance is crucial for ensuring patient safety and optimizing galsulfase treatment efficacy.
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