US FDA Approves Acadia Pharmaceuticals’ DAYBUE to Treat Rett Syndrome


Acadia Pharmaceuticals, Inc. (ACAD) announced that DAYBUE (trofinetide), a treatment for Rett syndrome, has been approved by the US FDA for use in adults and children over the age of two. The first and only drug that has been approved for the treatment of Rett syndrome is DAYBUE.

This day marks a significant turning point for Acadia and the Rett community. As the principal FDA-endorsed drug for the treatment of Rett condition, DAYBUE presently offers the possibility to cause significant contrasts in the existences of patients and their families who will have needed choices to treat the different and crippling cluster of side effects brought about by Rett disorder, said Steve Davis, Acadia’s CEO.

Steve Davis added that, they are thankful to all of the Rett syndrome patients, caregivers, clinical investigators, and employees who have helped to make today a reality, and they anticipate delivering DAYBUE to patients as soon as possible.

Jeffrey L. Neul, M.D., Ph.D., Professor of Pediatrics, Division of Neurology, Pharmacology, and Special Education, Vanderbilt University Medical Center said that, 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 a patient’s life.

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Presently, interestingly following quite a while of clinical exploration, medical care suppliers at last have a therapy choice to address a scope of center conduct, correspondence and actual side effects for their patients living with Rett condition.

A MECP2 gene mutation typically results in the complex, rare neurodevelopmental disorder known as Rett syndrome. It is characterized by a period of normal development from six to 18 months of age, after which there is significant developmental regression, including the loss of acquired communication skills and the ability to use the hands for purpose.