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

·1 min read
  • The FDA has approved Acadia Pharmaceuticals Inc's (NASDAQ: 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.

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