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# Acadia Drug for Rett Syndrome Approved
- URL: https://www.fdaweb.com/acadia-drug-for-rett-syndrome-approved/
- Published: 2023-03-13T12:00:00.000Z
- Updated: 2026-09-14T18:19:11.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5153963

FDA has approved an Acadia Pharmaceuticals NDA for Daybue (trofinetide) oral solution for treating Rett syndrome, a rare, genetic neurological and developmental (XXX INSERT XXX)disorder, in adults and children two years of age and older. The disorder affects brain development, according to an [FDA release](https://lnks.gd/l/eyJhbGciOiJIUzI1NiJ9.eyJidWxsZXRpbl9saW5rX2lkIjoxMDEsInVyaSI6ImJwMjpjbGljayIsImJ1bGxldGluX2lkIjoiMjAyMzAzMTMuNzMyMzk3NTEiLCJ1cmwiOiJodHRwczovL3d3dy5mZGEuZ292L2RydWdzL25ld3MtZXZlbnRzLWh1bWFuLWRydWdzL2ZkYS1hcHByb3Zlcy1maXJzdC10cmVhdG1lbnQtcmV0dC1zeW5kcm9tZT91dG1fbWVkaXVtPWVtYWlsJnV0bV9zb3VyY2U9Z292ZGVsaXZlcnkifQ.%5FgcsduwfAHCQsorCODJfXy0n-7VxpOjoOHXgQFC%5Fh94/s/1255931999/br/156045506190-l?ref=fdaweb.com), adding that patients typically experience a progressive loss of motor skills and language.

Approval was based on data from a 12-week placebo-controlled study (Study 1; NCT04181723). “The co-primary efficacy measures were change from baseline in the Rett Syndrome Behavior Questionnaire (RSBQ) total score and the Clinical Global Impression-Improvement (CGI-I) score at week 12,” FDA says. “Patients treated with Daybue demonstrated a statistically significant difference compared to placebo on the co-primary efficacy endpoints, as measured by the change from baseline in the RSBQ total score and the CGI-I score at week 12.”

The agency says that the most common adverse reactions included diarrhea (81%) and vomiting (27%).