The FDA approval of DAYBUE was based on the LAVENDER phase 3 study that evaluated the efficacy and safety in comparison to a placebo in 187 female patients


DAYBUE (trofinetide) is claimed as the first and only drug approved for the treatment of Rett syndrome. (Credit: Business Wire)

Acadia Pharmaceuticals has received approval from the US Food and Drug Administration (FDA) for DAYBUE (trofinetide) to treat Rett syndrome in adult and paediatric patients aged two years and above.

DAYBUE is a synthetic version of tripeptide glycine-proline-glutamate (GPE), a naturally occurring molecule.

Trofinetide is claimed as the first and only drug approved for the treatment of Rett syndrome, a complex, rare neurodevelopmental disorder.

Acadia Pharmaceuticals CEO Steve Davis said: “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.”

The FDA approval was based on the findings from the LAVENDER phase 3 trial.

The late-stage study was designed to assess the efficacy and safety of trofinetide versus placebo in 187 female patients with Rett syndrome aged five to 20 years.

As per the results, the patients treated with the investigational therapy showed statistically significant improvement compared to placebo on both co-primary efficacy endpoints.

The biopharmaceutical company said that the co-primary efficacy endpoints were measured by the change in Rett syndrome behaviour questionnaire (RSBQ) total score and the clinical global impression-improvement (CGI-I) scale score from baseline at week 12.

The Rett syndrome therapy is expected to be available in the US by the end of April 2023.

In 2018, Acadia Pharmaceuticals signed an exclusive license agreement with Neuren Pharmaceuticals to develop and commercialise trofinetide for the treatment of Rett syndrome and other indications in North America.