FDA Approves First Treatment for Rett Syndrome

TUESDAY, March 14, 2023 (HealthDay News) – The U.S. Food and Drug Administration has approved Daybue (trofinetide) for the treatment of Rett syndrome in adult and pediatric patients aged 2 years and older.

Approval was based on results from the phase 3 LAVENDER study in which the efficacy and safety of Daybue was assessed versus placebo in 187 female patients with Rett syndrome (ages 5 to 20 years). Compared with placebo, Daybue was associated with statistically significant improvement for change from baseline in the Rett Syndrome Behaviour Questionnaire total score and the Clinical Global Impression-Improvement scale score at week 12.

The most common side effects were diarrhea (82 percent) and vomiting (29 percent). Daybue is expected to be available in the United States by the end of April 2023.

"As the first FDA-approved drug for the treatment of Rett syndrome, Daybue now offers the potential to make meaningful differences in the lives of patients and their families who have lacked options to treat the diverse and debilitating array of symptoms caused by Rett syndrome," Steve Davis, CEO of Acadia Pharmaceuticals, said in a statement.

Approval of Daybue was granted to Acadia Pharmaceuticals.

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