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FDA Approves Novel Tx for Rare Rett Syndrome – Daybue

Last week the FDA approved a new oral solution therapy, Daybue (trofinetide) from Acadia Pharmaceuticals Inc., for the treatment of Rett syndrome in adult and pediatric patients two years of age and older.

Rett syndrome is a rare and severe neurodevelopmental disorder that occurs primarily in girls with onset as early as 1-2 years of age. Rett syndrome is believed to affect 6,000 to 9,000 patients in the United States. Only about 4,500 U.S. patients have been diagnosed. Symptoms range from loss of speech, mobility, and muscle tone as well as seizures, breathing problems, slowed growth, and near-constant hand movement.

Although approval did not carry a black box warning, the approval did include warnings and precautions for diarrhea and vomiting which may be particularly risky for the youngest patients.

Acadia did not confirm price for Daybue; however, analysts estimate a launch price of $450,000 annually. Given the small number of potential patients in the US, its cost, and side effect profile, it is expected that Daybue will launch through limited distribution.

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U.S. FDA approves Acadia’s genetic Rett syndrome drug

March 10 (Reuters) – The U.S. Food and Drug Administration approved Acadia Pharmaceuticals Inc’s drug for the treatment of Rett syndrome, a genetic brain disorder, the company said on Friday, making it the first approved drug for the condition.

The U.S. health regulator’s decision allows use of the trofinetide, to be sold under the brand name Daybue, in adult and pediatric patients two years of age and older and comes with a warning of diarrhea and weight loss.

The approval comes months after the FDA declined to approve expanded use of Acadia’s drug Nuplazid to treat psychosis related to Alzheimer’s disease. Analysts have said approval of Daybue would help drive growth for the company in the near term.

“We have put a lot of planning into potential commercialization of trofinetide, including resources for patients to access the drug,” said Acadia senior executive Kathie Bishop ahead of the approval.

Acadia forecasts sales of Nuplazid – its only drug on the market – of between $520 and $550 million this year, above analysts’ median expectations of $532.8 million, according to Refinitiv data. With the drugmaker facing a loss of exclusivity for Nuplazid in 2028, investors have pinned their hopes on a successful trofinetide launch.

After the FDA declined to approve the expanded use of Nuplazid, Acadia said it would not pursue that indication for Nuplazid further. The drugmaker plans to focus its resources on late-stage development of Nuplazid to treat symptoms of schizophrenia and early-stage development of another candidate, ACP-204, for Alzheimer’s-related psychosis.

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