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Acadia Pharmaceuticals Submits New Drug Application to the U.S. FDA for Trofinetide for the Treatment of Rett Syndrome

Tuesday, July 19, 2022

Acadia Pharmaceuticals Inc., announced that the company has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for trofinetide for the treatment of Rett syndrome in adults and pediatric patients two years of age and older.

“This is an important step forward for members of the Rett community who face a devastating disease with no approved therapies,” said Steve Davis, Chief Executive Officer. “We are grateful to the patients, their families and the physicians who have participated in the trofinetide clinical studies, including our pivotal Phase 3 Lavender study. We look forward to working with the FDA as it evaluates the NDA.”

The NDA submission is supported by results from the pivotal Phase 3 Lavender™ study evaluating the efficacy and safety of trofinetide versus placebo in 187 girls and young women aged 5-20 years with Rett syndrome. The study demonstrated a statistically significant improvement over placebo on the co-primary endpoints, the Rett Syndrome Behaviour Questionnaire (RSBQ) change from baseline to 12 weeks (p=0.0175; effect size=0.37) and the Clinical Global Impression-Improvement (CGI-I) scale score (p=0.0030; effect size=0.47) at 12 weeks. The RSBQ is a caregiver assessment of the core symptoms of Rett syndrome and the CGI-I is a global physician assessment of worsening or improving of Rett syndrome. In addition, the study also met its key secondary endpoint, the Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist–Social composite score (CSBS-DP-IT–Social) change from baseline to week 12 (p=0.0064; effect size=0.43), a caregiver scale of the ability to communicate.

In 2018, Acadia entered into an exclusive license agreement with Neuren Pharmaceuticals Limited (ASX: NEU) for the development and commercialization of trofinetide for the treatment of Rett syndrome and other indications in North America. Trofinetide has been granted Fast Track Status and Orphan Drug Designation for the treatment of Rett syndrome in the United States. An NDA with Orphan Drug Designation is eligible for priority review. Trofinetide has also been granted Rare Pediatric Disease (RPD) designation by the FDA. With such designation, Acadia expects to be awarded a Priority Review Voucher if the NDA is approved.

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