Library · Brain & nerves · Rett syndrome treatment
Trofinetide
Daybue
FDA-approved in 2023 as the first drug for Rett syndrome in patients two and older, this synthetic analogue of the IGF-1-derived tripeptide GPE showed statistically significant benefit on co-primary endpoints in the Phase 3 LAVENDER trial, through an unclear mechanism thought to involve neuroprotection.
Research score 88/100. Reference only, not a dose.
- Neuroprotection
- Anti-inflammatory
- US status
- Prescription Only
- Approval
- FDA Approved (2023)
- Evidence
- LAVENDER Phase 3: statistically significant improvement on both co-primary endpoints vs placebo
- Research score
- 88 / 100
- Indication
- Rett syndrome
- Origin
- United States
- Source category
- Brain & Nervous System
- Status group
- Approved / prescription
Mechanism
Believed to reduce inflammation, glutamate-driven excitotoxicity, oxidative stress, and cell death, though the precise mechanism is unknown.
Safety file
Most common adverse effects are diarrhea, about 82%, and vomiting, about 29%.
- Diarrhea (82%)
- Vomiting (29%)
- Fever
- Seizure
- Anxiety
- Decreased appetite
- Fatigue