trofinetide
trofinetide: developed by 1 company · 4 clinical trials tracked · 2 FDA decisions.
trofinetide evidence translation
What we can confirm
2 FDA records · 4 tracked trials
What it means
What to watch
Prepared research question
How could trofinetide → ACAD affect its developer, and what evidence supports the link?
Traceable citations · Unknowns marked
See linked entities and source evidence1 indications · 0 study sponsors
Related indications
Study sponsors
No additional listed-company sponsor is linked.
Development Pipeline (1)
| Company | Phase |
|---|---|
| ACADIA PHARMACEUTICALS INC (ACAD) | Phase 3 |
Clinical Trials (4)
An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome
Primary endpoint: Safety and Tolerability of Treatment With Oral Trofinetide
An Open-Label Extension Study of Continuing Treatment With Trofinetide for Rett Syndrome
Primary endpoint: Percentage of Patients With Treatment-emergent Adverse Events (TEAEs), With Serious Adverse Events (SAEs), and With Withdrawals Due to AEs
A 40-Week, Open-label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome
Primary endpoint: Percentage of Subjects With Treatment-emergent Adverse Events (TEAEs), Percentage of Subjects With Serious Adverse Events (SAEs), and Percentage of Subjects With Withdrawals Due to AEs
A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome
Primary endpoint: Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score - Change From Baseline to Week 12
FDA Decisions2 records
Frequently asked questions
Who is developing trofinetide?
trofinetide is being developed by ACADIA PHARMACEUTICALS INC.
How many clinical trials involve trofinetide?
BioSniper tracks 4 clinical trials involving trofinetide.
What is trofinetide's latest FDA decision?
The most recent tracked FDA decision for trofinetide is Approval for 1 INDICATIONS AND USAGE DAYBUE and DAYBUE STIX are indicated for the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older. DAYBUE and DAYBUE STIX are indicated for the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older. ( 1 ), dated December 11, 2025.