(Reuters) – The U.S. Food and Drug Administration (FDA) said on Monday it had approved a drug from Acadia Pharmaceuticals Inc (ACAD.O) to treat Rett syndrome, an inherited brain disorder.
A decision by US health officials allows adult and pediatric patients 2 years of age and older to use trophinetide marketed under the brand name Daybue with a warning of diarrhea and weight loss.
The approval comes months after the FDA denied approval to expand the use of Acadia’s drug Nuplazid to treat psychosis associated with Alzheimer’s disease. Analysts say Daybue’s approval will help boost the company’s growth in the short term.
“We have made a lot of plans for the potential commercialization of trophinetide, including resources for patient access to this drug,” said Cathy Bishop, Acadia’s senior executive, ahead of the approval. rice field.
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Acadia said it plans to make the drug available to patients by the end of April. Pricing details were not disclosed.
Prior to approval, SMBC Nikko Securities analyst David Huang estimated the list price at launch to be $450,000 per year. He predicts that by 2035 peak sales of trophinetide in the US will be $487.2 million. RBC Capital Markets analyst Gregory Renza also wrote before approval that peak U.S. sales he predicted would top $500 million by 2032, with an average annual launch price of about $425,000. I’m here.
Refinitiv data show that Acadia expects sales of the only drug on the market, nuplazid, to be between $520 million and $550 million this year, beating the median analyst estimate of $500 million. Over $32.8 million. Investors are pinning their hopes on a successful launch of trophinetide as the pharmaceutical company faces losing his Nuplazid exclusivity in 2028.
After the FDA denied approval for expanded use of Nuplazid, Acadia said it would no longer pursue that indication for Nuplazid. The pharmaceutical company plans to focus resources on late-stage development of his Nuplazid, which treats symptoms of schizophrenia, and early development of his ACP-204, another candidate for Alzheimer’s-related psychosis.
Rett syndrome is a rare neurodevelopmental disorder that occurs primarily in girls. According to government estimates, fewer than 50,000 people are affected in the United States.
Shares of the California-based company closed down 0.68% on Friday.
Acadia’s drug acts as an artificial form of the insulin-like growth factor IGF-1, helping to transmit nerve impulses as well as reduce inflammation in nerve tissue.
Acadia’s marketing authorization application was based on data from late-stage trials that showed treatment with this drug improved core symptoms of the disease compared to placebo. Symptom improvement was measured according to the rating scales Rett Syndrome Behavioral Questionnaire and Clinical Global Impression of Improvement.
The company had licensed the drug for development and marketing in North America in 2018 for an upfront payment of $10 million from Australian pharmaceutical company Neuron Pharmaceuticals (NEU.AX).
Reported by Bambi Satya, Nandini Srinivasan and Anirudh Saligrama of Bengaluru. Edited by Shailesh Kuber and William Mallard
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