FDA approves trofinetide for treatment of Rett syndrome


The FDA has approved trofinetide (Daybue; Acadia Pharmaceuticals) for treating Rett syndrome in patients aged 2 years and older.

Trofinetide (Daybue; Acadia Pharmaceuticals) has been approved by the FDA for use in patients aged 2 years and older with Rett syndrome.1

Rett syndrome is a genetic neurological and developmental disorder affecting about 1 in 10,000 female patients, and even fewer male patients. The disease impacts brain development, leading to a progressive loss of language and motor skills in patients.

Symptoms often begin to appear in patients aged 6 months, making them lose abilities they had developed in the early months of life. These abilities include crawling, walking, communicating, and using their hands. Babies will often lose these functions while aged 6 to 18 months, affecting their capabilities in speaking, walking, eating, and breathing.

“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, MD, PhD.2

Efficacy was tested in trofinetide during a randomized, double-blind, placebo-controlled, 12-week study.1 Participants aged 5 to 20 years with Rhett syndrome were given either trofinetide or placebo over the course of the study period, with dosages of trofinetide based on patient weight.

A change in the Rett Syndrome Behavior Questionnaire (RSBQ) total score and Clinical Global Impression-Improvement (CGI-I) score from baseline to week 12 was used to measure efficacy. A lower RSBQ score indicated decreased severity in symptoms and a lower CG-I score indicated improvement.

At week 12, significant improvements were seen in patients taking trofinetide compared to placebo. Common adverse reactions to trofinetide included diarrhea and vomiting. 

Trofinetide is the first drug approved for treatment of Rett syndrome, having received Orphan Drug, Priority Review, and Fast Track Drug Designations. Acadia Pharmaceuticals expects trofinetide to be available in the United States by the end of April 2023.2


1. FDA approves first treatment for Rett Syndrome. FDA. March 13, 2023. Accessed March 13, 2023. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-first-treatment-rett-syndrome?utm_medium=email&utm_source=govdelivery

2. Acadia Pharmaceuticals announces US FDA Approval of DAYBUE (trofinetide) for the treatment of rettsyndrome in adult and pediatric patients two years of age and older. Acadia. March 10, 2023. Accessed March 13, 2023. https://acadia.com/media/news-releases/acadia-pharmaceuticals-announces-u-s-fda-approval-of-daybue-trofinetide-for-the-treatment-of-rett-syndrome-in-adult-and-pediatric-patients-two-years-of-age-and-older/

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