Acadia Pharmaceuticals Inc. ACAD has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for trofinetide for the treatment of Rett syndrome.
The 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.
Steve Davis, Chief Executive Officer, commented : ”This is an important step forward for members of the Rett community who face a devastating disease with no approved therapies,. 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.”
Acadia’s lead candidate Trofinetide has been granted Fast Track Status, Orphan Drug Designation and Rare Pediatric Disease (RPD) designation for the treatment of Rett syndrome in the United States.
The company expected to be awarded a Priority Review coucher if the NDA is approved. An NDA with Orphan Drug Designation is eligible for priority review.
Price Action : Acadia shares are trading down at $15 on Monday during after-hours session.
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