FDA approves Acadia’s Daybue as first-ever treatment for Rett Syndrome

Acadia Pharmaceuticals announced that its drug, Daybue, has been approved by the US Food and Drug Administration (FDA) for the treatment of Rett Syndrome, a rare genetic brain disorder that primarily affects girls.

This marks the first approved drug for the condition.

The FDA’s decision allows the use of Daybue in adult and pediatric patients aged two years and above, with a warning of diarrhea and weight loss. Acadia plans to make the drug available to patients by the end of April, but did not disclose the price.

Before the approval, analysts predicted a list price of $450,000 per year and peak U.S. sales of over $500 million by 2032.

Acadia’s only drug on the market, Nuplazid, is estimated to achieve sales of between $520 million and $550 million this year, but the drug is facing a loss of exclusivity in 2028.

The approval of Daybue could help drive growth for the company in the near term. The drug acts as an artificial form of the insulin-like growth factor IGF-1 and helps to reduce inflammation in the nervous tissue as well as aid in the transmission of nerve impulses.