FDA accepts new drug application for Rett syndrome treatment

The FDA has accepted a new drug application from Acadia Pharmaceuticals Inc. for trofinetide, a synthetic analog of the amino‐terminal tripeptide of IGF-1 designed to treat core symptoms of Rett syndrome.

According to a release from Acadia, the NDA is supported by results from the phase 3 Lavender study, which evaluated the safety and efficacy of trofinetide compared with placebo in 187 girls and young women aged 5 to 20 years with Rett syndrome.

The study demonstrated a statistically significant improvement over placebo on co-primary endpoints, Rett Syndrome Behavior Questionnaire total score change from baseline to 12 weeks and Clinical Global Impression-Improvement scale score, the company said in the release. The study also met its secondary endpoint, a positive change in the Communication and Symbolic Behavior Scales Developmental Profile Infant-Toddler Checklist-Social Composite score from baseline to week 12, per the release.

“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,” Steve Davis, CEO at Acadia, said in the release. “This milestone reinforces Acadia’s ongoing commitment to advancing research into high unmet needs in disorders affecting the central nervous system.”

According to the release, the FDA has granted a priority review and assigned a Prescription Drug User Fee Act action date of March 12, 2023.