The U.S. Food and Drug Administration (FDA) has accepted for filing Acadia Pharmaceuticals' (NASDAQ:ACAD) New Drug Application (NDA) of trofinetide for the treatment of Rett syndrome.
The regulatory agency has granted a priority review and assigned a PDUFA action date of March 12, 2023.
The NDA submission is supported by data readout from the 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 Lavender study demonstrated a statistically significant improvement over placebo on the co-primary endpoints.
Steve Davis, Acadia's Chief Executive Officer, said, "We're pleased that the FDA has accepted our NDA filing and we will be working closely with them to facilitate completion of the review in a timely manner. If approved, trofinetide will be the first drug available for the treatment of Rett syndrome, a rare and devastating condition for patients and their families. This milestone reinforces Acadia's ongoing commitment to advancing research into high unmet needs in disorders affecting the central nervous system."
The agency has also informed the company that at this time they are not planning to hold an Advisory Committee meeting.
Trofinetide has been granted Fast Track Status, Orphan Drug Designation and Rare Pediatric Disease (RPD) designation by the FDA.
Rett syndrome is a rare genetic neurodevelopmental disorder that occurs primarily in females following a near normal development in the first two years of life.
Price Action : Acadia shares are trading around 1 percent higher at $17.60 on Monday during pre-market session.