Exploring adverse events associated with vosoritide monotherapy: Insights from the FDA Adverse Event Reporting System.

Li X, Luo X, Liu F, Wang C, Li X, Yu H

PloS one29 Jan 2026

PMID41610120PMCIDPMC12854448DOI10.1371/journal.pone.0341323PepBankPUB-41610120

Summary

BackgroundDwarfism, a condition characterized by short stature, has been the focus of therapeutic advancements with the emergence of novel peptide drugs. Vosoritide, indicated for certain types of dwarfism, has shown therapeutic potential in clinical trials. However, a comprehensive safety profile is essential for its clinical application. The current literature lacks a detailed assessment of vosoritide's safety, indicating a significant gap that this study aims to address.MethodsWe conducted a retrospective pharmacovigilance study by analyzing the FDA Adverse Event Reporting System (FAERS) database to evaluate adverse events (AEs) associated with vosoritide monotherapy. The study employed a case/non-case methodology and applied signal detection algorithms, including the Reporting Odds Ratio (ROR), Proportional Reporting Ratio (PRR), Bayesian Confidence Propagation Neural Network (BCPNN), and Multi-Item Gamma Poisson Shrinker (MGPS), to identify AE signals related to vosoritide use.ResultsThe study included 9319269 reports from the FAERS database, covering the period from 2022 to 2023, with 274 reports specifically citing vosoritide. A significant number of AEs were identified, with a notable incidence in the pediatric population. The most frequently reported AEs were related to endocrine disorders, including altered growth hormone levels and glucose homeostasis issues. Other affected system organ classes (SOCs) were infections and infestations, as well as skin and subcutaneous tissue disorders. Specific preferred terms (PTs) associated with vosoritide included "growth deceleration," "glycemic dysregulation," and "local injection site reactions."ConclusionThe findings from this study underscore the importance of close monitoring of vosoritide treatment, particularly in pediatric patients. The identification of both expected and unexpected AEs highlights the necessity for ongoing pharmacovigilance and further research to fully understand the safety profile of vosoritide in clinical practice. This study contributes to the broader field by emphasizing the critical need for patient safety considerations in the development of new therapeutic agents.

CC BYText from Europe PMC. Highlighted sentences are the source of results.

Results from this study5

5 results from studies that gave the peptide. 5 results from people.

01HumanObservational studySafety outcome
Among 274 FAERS reports citing vosoritide, adverse events showed notable reporting in the pediatric population.
In plain wordsVosoritide monotherapy reports included notable adverse event reporting in children.
Peptide
Vosoritide
Outcome
Not applicablePediatric adverse event reporting
Study
n: not reported in source
Outcome group
Adverse events · Whole body
Studied dose
Dose not stated
Source sentences
Summary
A significant number of AEs were identified, with a notable incidence in the pediatric population.
3 context sentences(design, population, supporting numbers)
Summary · context
We conducted a retrospective pharmacovigilance study by analyzing the FDA Adverse Event Reporting System (FAERS) database to evaluate adverse events (AEs) associated with vosoritide monotherapy.
Summary · context
The study employed a case/non-case methodology and applied signal detection algorithms, including the Reporting Odds Ratio (ROR), Proportional Reporting Ratio (PRR), Bayesian Confidence Propagation Neural Network (BCPNN), and Multi-Item Gamma Poisson Shrinker (MGPS), to identify AE signals related to vosoritide use.
Summary · context
The study included 9319269 reports from the FAERS database, covering the period from 2022 to 2023, with 274 reports specifically citing vosoritide.
Show in the study text Report a problemFND-4161012001
02HumanObservational studySafety outcome
In FAERS reports of vosoritide monotherapy, endocrine disorders, including altered growth hormone levels and glucose homeostasis issues, were most frequently reported.
In plain wordsVosoritide reports most frequently described endocrine problems, including altered growth hormone levels and glucose regulation.
Peptide
Vosoritide
Outcome
Not applicableEndocrine adverse event reporting
Study
n: not reported in source
Outcome group
Adverse events · Endocrine and hormones
Studied dose
Dose not stated
Source sentences
Summary
The most frequently reported AEs were related to endocrine disorders, including altered growth hormone levels and glucose homeostasis issues.
3 context sentences(design, population, supporting numbers)
Summary · context
We conducted a retrospective pharmacovigilance study by analyzing the FDA Adverse Event Reporting System (FAERS) database to evaluate adverse events (AEs) associated with vosoritide monotherapy.
Summary · context
The study employed a case/non-case methodology and applied signal detection algorithms, including the Reporting Odds Ratio (ROR), Proportional Reporting Ratio (PRR), Bayesian Confidence Propagation Neural Network (BCPNN), and Multi-Item Gamma Poisson Shrinker (MGPS), to identify AE signals related to vosoritide use.
Summary · context
The study included 9319269 reports from the FAERS database, covering the period from 2022 to 2023, with 274 reports specifically citing vosoritide.
Show in the study text Report a problemFND-4161012002
03HumanObservational studySafety outcome
In FAERS reports of vosoritide monotherapy, infections and infestations were among affected system organ classes.
In plain wordsVosoritide monotherapy reports included infections and infestations.
Peptide
Vosoritide
Outcome
Not applicableInfections and infestations reporting
Study
n: not reported in source
Outcome group
Adverse events · Infection
Studied dose
Dose not stated
Source sentences
Summary
Other affected system organ classes (SOCs) were infections and infestations, as well as skin and subcutaneous tissue disorders.
3 context sentences(design, population, supporting numbers)
Summary · context
We conducted a retrospective pharmacovigilance study by analyzing the FDA Adverse Event Reporting System (FAERS) database to evaluate adverse events (AEs) associated with vosoritide monotherapy.
Summary · context
The study employed a case/non-case methodology and applied signal detection algorithms, including the Reporting Odds Ratio (ROR), Proportional Reporting Ratio (PRR), Bayesian Confidence Propagation Neural Network (BCPNN), and Multi-Item Gamma Poisson Shrinker (MGPS), to identify AE signals related to vosoritide use.
Summary · context
The study included 9319269 reports from the FAERS database, covering the period from 2022 to 2023, with 274 reports specifically citing vosoritide.
Show in the study text Report a problemFND-4161012003
04HumanObservational studySafety outcome
In FAERS reports of vosoritide monotherapy, skin and subcutaneous tissue disorders were among affected system organ classes.
In plain wordsVosoritide monotherapy reports included skin and subcutaneous tissue disorders.
Peptide
Vosoritide
Outcome
Not applicableSkin and subcutaneous tissue disorder reporting
Study
n: not reported in source
Outcome group
Adverse events · Skin and wound
Studied dose
Dose not stated
Source sentences
Summary
Other affected system organ classes (SOCs) were infections and infestations, as well as skin and subcutaneous tissue disorders.
3 context sentences(design, population, supporting numbers)
Summary · context
We conducted a retrospective pharmacovigilance study by analyzing the FDA Adverse Event Reporting System (FAERS) database to evaluate adverse events (AEs) associated with vosoritide monotherapy.
Summary · context
The study employed a case/non-case methodology and applied signal detection algorithms, including the Reporting Odds Ratio (ROR), Proportional Reporting Ratio (PRR), Bayesian Confidence Propagation Neural Network (BCPNN), and Multi-Item Gamma Poisson Shrinker (MGPS), to identify AE signals related to vosoritide use.
Summary · context
The study included 9319269 reports from the FAERS database, covering the period from 2022 to 2023, with 274 reports specifically citing vosoritide.
Show in the study text Report a problemFND-4161012004
05HumanObservational studySafety outcome
FAERS reports linked vosoritide monotherapy with growth deceleration, glycemic dysregulation, and local injection site reactions.
In plain wordsVosoritide monotherapy reports included growth slowing, glucose regulation problems, and local injection site reactions.
Peptide
Vosoritide
Outcome
Direction not reportedSpecific preferred adverse event terms
Study
n: not reported in source
Comparator
Other reports in the FAERS database (case/non-case disproportionality analysis)
Outcome group
Adverse events · Whole body
Studied dose
Dose not stated
Source sentences
Summary
Specific preferred terms (PTs) associated with vosoritide included "growth deceleration," "glycemic dysregulation," and "local injection site reactions."
3 context sentences(design, population, supporting numbers)
Summary · context
We conducted a retrospective pharmacovigilance study by analyzing the FDA Adverse Event Reporting System (FAERS) database to evaluate adverse events (AEs) associated with vosoritide monotherapy.
Summary · context
The study employed a case/non-case methodology and applied signal detection algorithms, including the Reporting Odds Ratio (ROR), Proportional Reporting Ratio (PRR), Bayesian Confidence Propagation Neural Network (BCPNN), and Multi-Item Gamma Poisson Shrinker (MGPS), to identify AE signals related to vosoritide use.
Summary · context
The study included 9319269 reports from the FAERS database, covering the period from 2022 to 2023, with 274 reports specifically citing vosoritide.
Show in the study text Report a problemFND-4161012005

AI wrote and checked these results. AI can make mistakes, so check important results against the source.

Version history

PepBank keeps every version of the data on this study. Status changes from Retraction Watch, Europe PMC and Crossref are listed with their source.

Version 2Current
28 Oct 2026, 02:03 UTC
Study results updated
Version 1
27 Oct 2026, 16:37 UTC
First added · source update from Europe PMC

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