Drug updated on 4/17/2024
| Dosage Form | Solution (oral; 200 mg/mL) |
| Drug Class | Glycine–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.
Summary
- 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 Title | Year | Source |
|---|---|---|
| Daybue (trofinetide) Prescribing Information. | 2023 | Acadia Pharmaceuticals Inc. San Diego, CA |
Randomized Controlled Trials
| Document Title | Sex Distribution | Year | Source |
|---|---|---|---|
| Trofinetide treatment demonstrates a benefit over placebo for the ability to communicate in Rett syndrome. | 187Subjects F: 100% M: 0% | 2024 | Pediatric Neurology |
| Trofinetide for the treatment of Rett syndrome: a randomized phase 3 study. | 187Subjects F: 100% M: 0% | 2023 | Nature Medicine |
| Design and outcome measures of LAVENDER, a phase 3 study of trofinetide for Rett syndrome. | 187Subjects F: 100% M: 0% | 2022 | Contemporary Clinical Trials |
Document Title
Sex Distribution:
F:100%
M:0%
187Subjects
Year:
2024
Source:Pediatric Neurology
Sex Distribution:
F:100%
M:0%
187Subjects
Year:
2023
Source:Nature Medicine
Document Title
Sex Distribution:
F:100%
M:0%
187Subjects
Year:
2022
Source:Contemporary Clinical Trials