On July 18, Acadia announced that it had submitted a marketing application for trofinetide to the FDA for the treatment of adults and pediatric patients with Rett syndrome over the age of 2.
Reiter syndrome is a rare inherited neurodevelopmental disorder caused by mutations in the X chromosome of a gene called MECP2.3, which often occurs in girls and typically does not show symptoms until 18 months of age . Rett syndrome causes severe impairment of central nervous system function, including loss of communication skills, purposeful use of hands, abnormal gait, and stereotyped hand movements such as twisting/squeezing, clapping/slapping, gargling and washing/ Friction automation. Patients with Rett syndrome may also experience a range of other symptoms, such as gastrointestinal complications, skeletal abnormalities, neuroendocrine abnormalities, disruptive and anxiety-like behaviors, as well as disturbances in mood regulation and sleep. Currently, there is no drug for Rett syndrome in the world.
Trofinetide, a novel synthetic analog of the insulin-like growth factor 1 (IGF-1) amino-terminal tripeptide, is thought to stimulate synaptic maturation, overcoming synaptic and neuronal immaturity characteristic of the pathophysiology of Rett syndrome, by Relieves neuroinflammation and supports the protection of synaptic function to treat the main clinical symptoms of Rett syndrome. It has obtained Fast Track designation and orphan drug designation granted by the FDA, as well as rare pediatric disease drug designation. If approved, it can receive a Priority Review Voucher.
The marketing application is primarily based on the results of the Phase III Lavender study. In 187 patients with Rett syndrome aged 5 to 20 years, trofinetide significantly improved the Rett Syndrome Behavioral Questionnaire (RSBQ,) and Clinical Outcome Global Scale (CGI-I) scores at week 12 compared with placebo.