US FDA Approves Rett Syndrome Drug by Acadia

HEALTHCARE

The US FDA granted Acadia Pharmaceuticals’ DAYBUE (trofinetide) approval for the treatment of Rett syndrome in adults and children over the age of two last Friday. DAYBUE is the first and only medication approved for the treatment of Rett syndrome.

Acadia and the Rett community have reached a significant turning point today. Steve Davis, CEO of Acadia, stated that DayBUE now offers the potential to significantly improve the lives of patients and their families who had no other treatment options for Rett syndrome symptoms. The FDA has approved it for the first time as a treatment for Rett syndrome.

Steve Davis stated that they look forward to delivering DAYBUE to patients as soon as possible and are thankful to all Rett syndrome patients, caregivers, clinical investigators, and their employees for their contributions to making today a reality.

A MECP2 gene mutation typically results in the complex, rare neurodevelopmental disorder known as Rett syndrome. Hand stereotypies, such as hand wringing and clapping, and abnormal gait may also be symptoms of Rett syndrome. Rett condition is approved to influence 6,000 to 9,000 patients in the United States, along with an analyzed population of roughly 4,500 US patients.

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The pivotal Phase III LAVENDER study, which compared the safety and efficacy of trofinetide to placebo in 187 female Rett syndrome patients aged five to twenty, supported the FDA’s approval of DAYBUE.