Trofinetide

(Daybue®)

Trofinetide

Drug updated on 4/17/2024

Dosage FormSolution (oral; 200 mg/mL)
Drug ClassGlycine–proline–glutamate (GPE) synthetic analogs
Ongoing and
Completed Studies
ClinicalTrials.gov

Indication

  • Indicated for the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older.

Latest News

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Summary
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  • Trofinetide (Daybue) is approved for the management of Rett syndrome in adults and pediatric patients 2 years of age and older, with substantial progress observed in comparison to a placebo in various clinical measurements across phase 3 studies.
  • Three studies from Randomized Controlled Trials were examined to collect information on the efficacy, safety, and comparison with other therapeutic options for this drug, including the types of populations involved during trials and subgroup considerations.
  • The LAVENDER study revealed that Trofinetide (Daybue) significantly enhances communication-related abilities compared to a placebo. This benefit was noted with effect sizes indicating moderate clinical relevance.
  • Common side effects like diarrhea were observed but were generally mild to moderate. A phase 2 study emphasized Trofinetide's tolerability at the highest dosage across three measures: RSBQ, CGI-I, RTT-DSC-VAS.
  • Although specific comparisons to other therapeutic options were not detailed in the studies examined, Trofinetide appears to offer significant benefits in managing core symptoms of Rett syndrome, particularly in improving communication, which is a major concern due to the lack of previously approved treatments for this condition.
  • The research primarily focused on female populations aged between 5 -20 years due to its prevalence among females. Significant improvements across communication scales suggest effectiveness across the spectrum, including both behavioral and functional aspects. This makes it a significant advancement in the therapeutic landscape, offering hope for improved outcomes and quality of life for those affected by this debilitating condition.

Product Monograph / Prescribing Information

Document TitleYearSource
Daybue (trofinetide) Prescribing Information. 2023Acadia Pharmaceuticals Inc. San Diego, CA

Randomized Controlled Trials

Document TitleSex DistributionYearSource
Trofinetide treatment demonstrates a benefit over placebo for the ability to communicate in Rett syndrome.
187Subjects
F: 100%
M: 0%
2024Pediatric Neurology
Trofinetide for the treatment of Rett syndrome: a randomized phase 3 study.
187Subjects
F: 100%
M: 0%
2023Nature Medicine
Design and outcome measures of LAVENDER, a phase 3 study of trofinetide for Rett syndrome.
187Subjects
F: 100%
M: 0%
2022Contemporary Clinical Trials

Sex Distribution:

F:100%
M:0%
187Subjects

Year:

2024

Source:Pediatric Neurology


Sex Distribution:

F:100%
M:0%
187Subjects

Year:

2023

Source:Nature Medicine


Sex Distribution:

F:100%
M:0%
187Subjects

Year:

2022

Source:Contemporary Clinical Trials