Is Trofinetide safe?
Extension studies; few hundred patients
In LAVENDER, 81 percent of trofinetide patients had diarrhea, versus 19 percent on placebo (82 versus 20 percent in the label's table). Vomiting affected 29 percent versus 12 percent. Adverse reactions made 19 percent of treated patients quit. Diarrhea alone made 15 percent quit. The label says to stop laxatives before starting. It also says to manage diarrhea actively. Weight loss is the other listed warning. 24
No new safety signals emerged in the extension studies. But experience is limited to 260 patients aged 2 to 40. The phase 3 trial covered ages 5 to 20. Use in children aged 2 to 4 rests on pharmacokinetic and safety data in 13 patients. Use in adults relies largely on extrapolation. Long-term effects on growth and development are not yet known. 34
Diarrhea, in about 81 percent of patients during the phase 3 trial. About a quarter had vomiting. About one in seven stopped because of it. Weight loss is the other labeled warning. 24
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Citations
- Berry-Kravis 2020, Pediatric Neurology: phase 2 trial of trofinetide in adolescent and adult males with fragile X syndrome doi.org
- Neul 2023, Nature Medicine: Trofinetide for the treatment of Rett syndrome, phase 3 LAVENDER trial doi.org
- FDA 2023: Daybue (trofinetide) approval label accessdata.fda.gov
- DailyMed: Daybue (trofinetide) current prescribing information dailymed.nlm.nih.gov
- Glaze 2017, Pediatric Neurology: double-blind randomized placebo-controlled phase 2 study of trofinetide in adolescent and adult females with Rett syndrome pubmed.ncbi.nlm.nih.gov
- Glaze 2019, Neurology: double-blind randomized placebo-controlled phase 2 study of trofinetide in pediatric Rett syndrome pubmed.ncbi.nlm.nih.gov
Reference information only, not medical advice. Drawn from the Trofinetide article, fact-checked against the cited sources. No dosing anywhere on this site.