Acadia Pharma's Rett Syndrome Drug Becomes First FDA-Approved Treatment

The FDA has approved Acadia Pharmaceuticals Inc's ACAD Daybue (trofinetide) for Rett syndrome in adult and pediatric patients two years of age and older.
Daybue is the first and only drug approved for treating Rett syndrome, a rare genetic neurological and developmental disorder that causes a progressive loss of motor skills and language. Rett syndrome primarily affects females.
Rett syndrome is believed to affect 6,000 to 9,000 patients in the U.S., with a diagnosed population of approximately 4,500 U.S. patients.
The FDA approval of Daybue was supported by results from the pivotal Phase 3 LAVENDER study.
In the study, treatment with Daybue demonstrated statistically significant improvement compared to placebo on both co-primary efficacy endpoints.
Also Read: Analysts See Steady Growth For Acadia Pharma's Nuplazid.
Daybue is expected to be available in the U.S. by the end of April 2023.
In 2018, Acadia entered into an exclusive license agreement with Neuren Pharmaceuticals Limited to develop and commercialize trofinetide for treating Rett syndrome and other indications in North America.
With the FDA approval of DAYBUE, Acadia has received a Rare Pediatric Disease Priority Review Voucher, which can be used to obtain priority review for a subsequent application.
Price Action: ACAD shares are down 6.73% at $19.00 on the last check Monday.