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Study · Observational study · n = 2,824 · 2026

11 results

Post-marketing safety concerns with trofinetide: a disproportionality analysis of the first therapeutic agent for Rett syndrome based on the FDA adverse event reporting system (FAERS).

Yu X, Zhong J, Lin Z, Fu H, Zhang Y

Frontiers in pharmacology14 Jan 2026Cited by 2 (Europe PMC)

PMID41613782 (opens in a new tab)PMCIDPMC12847357 (opens in a new tab)DOI10.3389/fphar.2026.1643906 (opens in a new tab)PepBankPUB-41613782
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Summary

ObjectiveRett syndrome (RTT) is a severe, rare and chronic disease that necessitates long-term treatment. In March 2023, trofinetide was approved by the US Food and Drug Administration as the first treatment for RTT. Because of the constraints of clinical trials, certain delayed and infrequent adverse events (AEs) may go unreported, especially in orphan disease. Further research is required to investigate the potential AE signals of trofinetide in real-world scenarios, identify rare and severe AEs associated with this treatment, and promote the safe use of trofinetide among RTT patients.MethodsThe data were extracted from the FAERS database from the first quarter of 2023 to the fourth quarter of 2024. Signal mining was conducted using frequency and Bayesian methods to identify positive signals associated with trofinetide. In order to obtain similar reports, the generic name "trofinetide" and commercial name "DAYBUE" were utilized.ResultsA total of 3,293,302 AE reports were collected, with 15,266 AE reports from 2,824 patients related to trofinetide designated as the primary suspected drug. All the reports were from the United States. Signal mining identified 25 system organ classes (SOCs), involving 155 preferred terms (PTs). Gastrointestinal disorders had the highest report count, with diarrhoea being the most prominent AE with high report numbers and signal strength. Several noteworthy AEs except for gastrointestinal disorders were identified which are not included on the drug label, such as nasopharyngitis, decreased appetite, weight decreased, gastroenteritis viral, influenza and irritability. Time-to-onset analysis shows that most AEs occurred in 0-30 days.ConclusionThis study unveils certain potential risks associated with trofinetide in real-world applications. Medical staffs should pay more attention to AEs of patients on the first month.
CC BYText from Europe PMC. Highlighted sentences are the source of results.

Results from this study11

11 results from studies that gave the peptide. 11 results from people.

  1. 01HumanObservational studySafety outcome

    In FAERS, trofinetide was the primary suspected drug in 15,266 adverse event reports from 2,824 patients.

    In plain wordsIn FAERS, trofinetide was the primary suspected drug in 15,266 adverse event reports from 2,824 patients.

    Peptide
    Trofinetide
    Outcome
    Not applicableTrofinetide-associated adverse event reports
    Study

    n = 2,824

    Outcome group
    Adverse events · Whole body
    Studied dose
    Dose not stated

    Measurements

    Adverse event reports
    15,266 reports
    trofinetide primary suspect
    Patients
    2824 patients
    trofinetide primary suspect

    Source sentences

    Summary
    A total of 3,293,302 AE reports were collected, with 15,266 AE reports from 2,824 patients related to trofinetide designated as the primary suspected drug.
    2 context sentences
    Summary · context
    The data were extracted from the FAERS database from the first quarter of 2023 to the fourth quarter of 2024.
    Summary · context
    Signal mining was conducted using frequency and Bayesian methods to identify positive signals associated with trofinetide.
  2. 02HumanObservational studySafety outcome

    In FAERS trofinetide reports, signal mining identified 25 system organ classes involving 155 preferred terms.

    In plain wordsIn FAERS trofinetide reports, signals covered 25 organ classes and 155 adverse event terms.

    Peptide
    Trofinetide
    Outcome
    Not applicableSignal distribution
    Study

    n: not reported in source

    Outcome group
    Adverse events · Whole body
    Studied dose
    Dose not stated

    Measurements

    System organ classes
    25 classes
    signal mining
    Preferred terms
    155 terms
    signal mining

    Source sentences

    Summary
    Signal mining identified 25 system organ classes (SOCs), involving 155 preferred terms (PTs).
    2 context sentences
    Summary · context
    The data were extracted from the FAERS database from the first quarter of 2023 to the fourth quarter of 2024.
    Summary · context
    Signal mining was conducted using frequency and Bayesian methods to identify positive signals associated with trofinetide.
  3. 03HumanObservational studySafety outcome

    In FAERS trofinetide reports, gastrointestinal disorders had the highest report count.

    In plain wordsIn FAERS trofinetide reports, gastrointestinal disorders had the highest report count.

    Peptide
    Trofinetide
    Outcome
    Not applicableGastrointestinal disorders
    Study

    n: not reported in source

    Outcome group
    Gastrointestinal adverse events · Gastrointestinal
    Studied dose
    Dose not stated

    Source sentences

    Summary
    Gastrointestinal disorders had the highest report count, with diarrhoea being the most prominent AE with high report numbers and signal strength.
    2 context sentences
    Summary · context
    The data were extracted from the FAERS database from the first quarter of 2023 to the fourth quarter of 2024.
    Summary · context
    Signal mining was conducted using frequency and Bayesian methods to identify positive signals associated with trofinetide.
  4. 04HumanObservational studySafety outcome

    In FAERS trofinetide reports, diarrhoea was the most prominent adverse event by report numbers and signal strength.

    In plain wordsIn FAERS trofinetide reports, diarrhea was the most prominent adverse event by report numbers and signal strength.

    Peptide
    Trofinetide
    Outcome
    Not applicableDiarrhoea signal
    Study

    n: not reported in source

    Outcome group
    Gastrointestinal adverse events · Gastrointestinal
    Studied dose
    Dose not stated

    Source sentences

    Summary
    Gastrointestinal disorders had the highest report count, with diarrhoea being the most prominent AE with high report numbers and signal strength.
    2 context sentences
    Summary · context
    The data were extracted from the FAERS database from the first quarter of 2023 to the fourth quarter of 2024.
    Summary · context
    Signal mining was conducted using frequency and Bayesian methods to identify positive signals associated with trofinetide.
  5. 05HumanObservational studySafety outcome

    In FAERS, trofinetide reports included a nasopharyngitis adverse event signal absent from the drug label.

    In plain wordsIn FAERS, trofinetide was linked to a nasopharyngitis signal absent from the drug label.

    Peptide
    Trofinetide
    Outcome
    Not applicableNasopharyngitis signal
    Study

    n: not reported in source

    Outcome group
    Adverse events · Infection
    Studied dose
    Dose not stated

    Source sentences

    Summary
    Several noteworthy AEs except for gastrointestinal disorders were identified which are not included on the drug label, such as nasopharyngitis, decreased appetite, weight decreased, gastroenteritis viral, influenza and irritability.
    2 context sentences
    Summary · context
    The data were extracted from the FAERS database from the first quarter of 2023 to the fourth quarter of 2024.
    Summary · context
    Signal mining was conducted using frequency and Bayesian methods to identify positive signals associated with trofinetide.
  6. 06HumanObservational studySafety outcome

    In FAERS, trofinetide reports included a decreased appetite adverse event signal absent from the drug label.

    In plain wordsIn FAERS, trofinetide was linked to a decreased appetite signal absent from the drug label.

    Peptide
    Trofinetide
    Outcome
    Not applicableDecreased appetite signal
    Study

    n: not reported in source

    Outcome group
    Adverse events · Metabolic and weight
    Studied dose
    Dose not stated

    Source sentences

    Summary
    Several noteworthy AEs except for gastrointestinal disorders were identified which are not included on the drug label, such as nasopharyngitis, decreased appetite, weight decreased, gastroenteritis viral, influenza and irritability.
    2 context sentences
    Summary · context
    The data were extracted from the FAERS database from the first quarter of 2023 to the fourth quarter of 2024.
    Summary · context
    Signal mining was conducted using frequency and Bayesian methods to identify positive signals associated with trofinetide.
  7. 07HumanObservational studySafety outcome

    In FAERS, trofinetide reports included a weight decreased adverse event signal absent from the drug label.

    In plain wordsIn FAERS, trofinetide was linked to a weight loss signal absent from the drug label.

    Peptide
    Trofinetide
    Outcome
    Not applicableWeight decreased signal
    Study

    n: not reported in source

    Outcome group
    Adverse events · Metabolic and weight
    Studied dose
    Dose not stated

    Source sentences

    Summary
    Several noteworthy AEs except for gastrointestinal disorders were identified which are not included on the drug label, such as nasopharyngitis, decreased appetite, weight decreased, gastroenteritis viral, influenza and irritability.
    2 context sentences
    Summary · context
    The data were extracted from the FAERS database from the first quarter of 2023 to the fourth quarter of 2024.
    Summary · context
    Signal mining was conducted using frequency and Bayesian methods to identify positive signals associated with trofinetide.
  8. 08HumanObservational studySafety outcome

    In FAERS, trofinetide reports included a viral gastroenteritis adverse event signal absent from the drug label.

    In plain wordsIn FAERS, trofinetide was linked to a viral gastroenteritis signal absent from the drug label.

    Peptide
    Trofinetide
    Outcome
    Not applicableViral gastroenteritis signal
    Study

    n: not reported in source

    Outcome group
    Adverse events · Infection
    Studied dose
    Dose not stated

    Source sentences

    Summary
    Several noteworthy AEs except for gastrointestinal disorders were identified which are not included on the drug label, such as nasopharyngitis, decreased appetite, weight decreased, gastroenteritis viral, influenza and irritability.
    2 context sentences
    Summary · context
    The data were extracted from the FAERS database from the first quarter of 2023 to the fourth quarter of 2024.
    Summary · context
    Signal mining was conducted using frequency and Bayesian methods to identify positive signals associated with trofinetide.
  9. 09HumanObservational studySafety outcome

    In FAERS, trofinetide reports included an influenza adverse event signal absent from the drug label.

    In plain wordsIn FAERS, trofinetide was linked to an influenza signal absent from the drug label.

    Peptide
    Trofinetide
    Outcome
    Not applicableInfluenza signal
    Study

    n: not reported in source

    Outcome group
    Adverse events · Infection
    Studied dose
    Dose not stated

    Source sentences

    Summary
    Several noteworthy AEs except for gastrointestinal disorders were identified which are not included on the drug label, such as nasopharyngitis, decreased appetite, weight decreased, gastroenteritis viral, influenza and irritability.
    2 context sentences
    Summary · context
    The data were extracted from the FAERS database from the first quarter of 2023 to the fourth quarter of 2024.
    Summary · context
    Signal mining was conducted using frequency and Bayesian methods to identify positive signals associated with trofinetide.
  10. 10HumanObservational studySafety outcome

    In FAERS, trofinetide reports included an irritability adverse event signal absent from the drug label.

    In plain wordsIn FAERS, trofinetide was linked to an irritability signal absent from the drug label.

    Peptide
    Trofinetide
    Outcome
    Not applicableIrritability signal
    Study

    n: not reported in source

    Outcome group
    Adverse events · Mental health and mood
    Studied dose
    Dose not stated

    Source sentences

    Summary
    Several noteworthy AEs except for gastrointestinal disorders were identified which are not included on the drug label, such as nasopharyngitis, decreased appetite, weight decreased, gastroenteritis viral, influenza and irritability.
    2 context sentences
    Summary · context
    The data were extracted from the FAERS database from the first quarter of 2023 to the fourth quarter of 2024.
    Summary · context
    Signal mining was conducted using frequency and Bayesian methods to identify positive signals associated with trofinetide.
  11. 11HumanObservational studySafety outcome

    In FAERS trofinetide reports, most adverse events occurred in 0-30 days.

    In plain wordsIn FAERS trofinetide reports, most adverse events occurred in 0-30 days.

    Peptide
    Trofinetide
    Outcome
    Not applicableAdverse event time to onset
    Study

    n: not reported in source

    Outcome group
    Adverse events · Whole body
    Studied dose
    Dose not stated

    Measurements

    Time to onset
    0 to 30 days
    most adverse events

    Source sentences

    Summary
    Time-to-onset analysis shows that most AEs occurred in 0-30 days.
    2 context sentences
    Summary · context
    The data were extracted from the FAERS database from the first quarter of 2023 to the fourth quarter of 2024.
    Summary · context
    Signal mining was conducted using frequency and Bayesian methods to identify positive signals associated with trofinetide.

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.

  1. Version 2Current

    28 Oct 2026, 02:03 UTC

    Study results updated

  2. Version 1

    27 Oct 2026, 16:37 UTC

    First added · source update from Europe PMC

Subjects

Terms as the source lists them.

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