Acadia submits NDA to FDA for trofinetide for treatment of Rett syndrome


If approved, trofinetide has the potential to be a new treatment for Rett syndrome in pediatric and adult patients 2 years and older.

Acadia Pharmaceuticals announced it has submitted a New Drug Application to the US Food and Drug Administration for trofinetide as a potential new treatment for Rett syndrome in pediatric and adult patients 2 years and older.

Rett syndrome is a rare genetic neurodevelopmental disorder that primarily affects females after a near-normal development in the first 2 years of being born and is caused by mutations in the X chromosome on gene MECP2. This disorder leads to significant impairment of the central nervous system, which includes purposeful hand use, gait abnormalities, loss of communication skills, and stereotypic hand movements.1

Trofinetide is an investigational, novel synthetic analog of the amino‐terminal tripeptide of IGF-1 intended to treat the main symptoms of Rett syndrome by reducing neuroinflammation and supporting synaptic function.

The submission is backed by positive results from Acadia's phase 3 Lavendar study, which investigated the safety and efficacy of trofinetide vs placebo in 187 girls and young women ages 5 to 20 years with Rett syndrome.

Results of the study demonstrated a statistically significant improvement over placebo on the co-primary endpoints. The co-primary endpoints included a change in baseline to 12 weeks in the Rett Syndrome Behaviour Questionnaire (RSBQ) (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 study also met its secondary endpoint, which was a change from baseline to week 12 in the Communication and Symbolic Behavior Scales Developmental Profil Infant-Toddler Checklist–Social composite score (CSBS-DP-IT–Social) (p=0.0064; effect size=0.43). This is a caregiver scale to evaluate the ability to communicate.

Trofinetide was previously granted Fast Track Status and Orphan Drug Designation for the treatment of Rett syndrome in the United States, as well as granted Rare Pediatric Disease (RPD) designation by the FDA. Acadia said it expects to be awarded a Priority Review Voucher from the FDA if the NDA is approved, according to a company press release.


1. Acadia pharmaceuticals submits new drug application to the US FDA for trofinetide for the treatment of rett syndrome. Business Wire. July 18, 2022. Accessed July 19, 2022.

Related Videos
Juanita Mora, MD
Rupa Wong, MD | Image Credit: Rupa Wong, MD
Tina Tan, MD, FAAP, FIDSA, FPIDS, editor in chief, Contemporary Pediatrics, professor of pediatrics, Feinberg School of Medicine, Northwestern University, pediatric infectious diseases attending, Ann & Robert H. Lurie Children's Hospital of Chicago
John Bradley, MD
FDA approves B-VEC to treat dystrophic epidermolysis bullosa patients 6 months and older | Image Credit: bankrx - Image Credit: bankrx -
Pfizer's infant RSV vaccine receives FDA Advisory Committee's support | Image Credit: Dr_Microbe - Image Credit: Dr_Microbe
© 2024 MJH Life Sciences

All rights reserved.