Acadia's Daybue wins FDA nod as first treatment for Rett syndrome

Doctors and scientists have been studying Rett syndrome for decades, but up until now, there have been no approved treatments. Enter Acadia's Daybue.

Friday, Acadia's new medicine gained an FDA approval to treat patients with Rett syndrome ages 2 and older. The approval marks the first for a treatment for the neurodevelopmental disorder. 

Rett doesn't only affect patients' ability to verbally communicate; it also hinders nonverbal communication. That means patients can’t speak with their voices, their hands or their eyes, leaving them “trapped in their own body,” Kathie Bishop, Ph.D., Acadia’s senior vice president and head of rare diseases, said in an interview.

The disease is associated with many other symptoms, including breathing issues, lack of motor function and seizures.

Patients in Acadia’s phase 3 Lavender trial reported treatment outcomes through a questionnaire that asked them about a range of common symptoms. With Daybue treatment, patients reported consistent benefits, especially in their ability to use eye gaze and their hands for communication, Bishop said.

Daybue, a liquid medicine, is dosed twice per day. Since Rett is a chronic condition, Bishop expects patients to take the therapy every day.  

Rett is mostly found in females and the drug's pivotal trial was conducted in solely female patients.  

Ahead of the approval, Acadia was busy educating parents of children with the disease. Within that group, the company will hone in on parents of newly diagnosed parents for the launch.

Acadia expects the drug to be available by the end of April.  

In the U.S., between 6,000 and 10,000 patients are estimated to have the disease. About 4,500 patients are already seeking treatment with a physician, the company found through its market research.  

In 2018, Acadia licensed the drug from Australia’s Neuren Pharmaceuticals, which retains rights to Daybue outside of North America.  

Besides Daybue, Acadia markets Parkinson's disease psychosis drug Nuplazid. Previously, that drug was at the center of a controversy after CNN highlighted hundreds of deaths for patients on the medicine. The Justice Department later investigated Acadia's marketing practices.

After a review, the FDA reaffirmed the drug's risk-benefit profile in a public statement. And in November 2020, Acadia said the DOJ concluded its probe without deciding to take any other actions.

Editor's note: This story was updated with the latest information about Acadia's Nuplazid.