On March 10, Acadia Pharmaceuticals announced that the U.S. FDA has approved Daybue (trofinetide) for the treatment of Rett syndrome in adult and pediatric patients two years of age and older. Daybue (trofinetide) is the first and only drug approved for the treatment of Rett syndrome.
Rett syndrome is a complex, rare, neurodevelopmental disorder typically caused by a genetic mutation on the MECP2 gene. It is characterized by a period of normal development until six to 18 months of age, followed by significant developmental regression with loss of acquired communication skills and purposeful hand use. Symptoms of Rett syndrome may also include development of hand stereotypies, such as hand wringing and clapping, and gait abnormalities. Rett syndrome is believed to affect 6,000 to 9,000 patients in the U.S., with a diagnosed population of approximately 4,500 U.S. patients.
Daybue (trofinetide) is a synthetic version of a naturally occurring molecule known as the tripeptide glycine-proline-glutamate (GPE). In 2018, Acadia entered into an exclusive license agreement with Neuren Pharmaceuticals Limited for the development and commercialization of trofinetide for the treatment of Rett syndrome and other indications in North America. Daybue is expected to be available in the U.S. by the end of April, 2023.
The FDA approval of Daybue was supported by results from the pivotal Phase 3 LAVENDER study evaluating the efficacy and safety of trofinetide versus placebo in 187 female patients with Rett syndrome five to 20 years of age.
In the study, treatment with Daybue demonstrated statistically significant improvement compared to placebo on both co-primary efficacy endpoints, as measured by the change from baseline in Rett Syndrome Behaviour Questionnaire (RSBQ) total score (p=0.018) and the Clinical Global Impression-Improvement (CGI-I) scale score (p=0.003) at week 12.
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