Acadia Pharmaceuticals Receives FDA Approval for First Treatment for Rare Genetic Disorder

On March 10, 2023, FDA approved US-based Acadia Pharmaceuticals’ Daybue (trofinetide), an oral solution for treating a rare genetic disease. According to an agency press release, the drug is the first treatment for Rett syndrome, a rare, genetic neurological disorder. The drug is indicated for treating adult patients and children two years of age or older who have the condition.

Rett syndrome affects the way the brain develops, and patients with this disorder experience a progressive loss of motor skills and language. A hallmark symptom of Rett syndrome is near constant repetitive hand movements, such as rubbing or clapping. The disorder, which primarily affects females (1 in 10,000) and even more rarely affects males, leads to severe impairments that affect nearly every aspect of life, including speaking, walking, eating, and breathing.

Daybue’s efficacy and safety were evaluated in a randomized, double-blind, placebo-controlled, 12-week study (Study 1; NCT04181723) involving patients with Rett syndrome at five to 20 years of age. Patients treated with the drug demonstrated a statistically significant difference compared to placebo on co-primary efficacy endpoints, according to the agency press release.

The drug is expected to be available in the United States by the end of April 2023, according to an Acadia Pharmaceuticals company press release. Acadia entered into an exclusive license agreement with Neuren Pharmaceuticals in 2018 for the development and commercialization of trofinetide in North America for the Rett syndrome indication as well as other indications.

“Rett syndrome is a profoundly debilitating and complex, rare, neurodevelopmental disorder that presents differently across patients and can lead to an array of unpredictable symptoms throughout the course of a patient’s life,” said Jeffrey L. Neul, Annette Schaffer Eskind chair and director, Vanderbilt Kennedy Center, professor of Pediatrics, Division of Neurology, Pharmacology, and Special Education, Vanderbilt University Medical Center, and Phase III study (LAVENDER) investigator, in the Acadia Pharmaceuticals press release. “Now, for the first time after decades of clinical research, healthcare providers finally have a treatment option to address a range of core behavioral, communication, and physical symptoms for their patients living with Rett syndrome.”

Source: FDA and Acadia Pharmaceuticals