Axsome Therapeutics Provides Update on the New Drug Application (NDA) for AXS-14 for the Management of Fibromyalgia

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NEW YORK, June 09, 2025 (GLOBE NEWSWIRE) — Axsome Therapeutics, Inc. (NASDAQ: AXSM), a biopharmaceutical company leading a new era in the treatment of central nervous system (CNS) disorders, today announced it has received a Refusal to File (RTF) letter from the U.S. Food and Drug Administration (FDA) for its New Drug Application (NDA) for AXS-14 (esreboxetine) for the management of fibromyalgia.

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The FDA states that upon preliminary review, it found that the NDA was not sufficiently complete to permit a substantive review. Specifically, the FDA does not consider the second of the two placebo-controlled trials in the submission to be adequate and well-controlled because its primary endpoint was at 8 weeks and it used a flexible-dose paradigm. The FDA indicated that the first of the two placebo-controlled trials in the submission, which utilized a 12-week endpoint and a fixed-dose paradigm, is adequate and well-controlled. The FDA did not raise any questions relating to the positive results of the studies, both of which met their primary endpoints.

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To address the FDA’s feedback, Axsome will conduct an additional controlled trial, which will use a fixed-dose paradigm and a 12-week primary endpoint as requested by the FDA. Axsome anticipates initiating this trial in the fourth quarter of 2025.

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“The clear feedback provided by the FDA’s Division of Anesthesiology, Addiction Medicine, and Pain Medicine allows us to move expeditiously with the continued development of this important investigational medicine for the approximately 17 million patients in the U.S. living with fibromyalgia. We are well positioned to initiate a new controlled trial that will incorporate the FDA’s feedback by the end of 2025,” said Herriot Tabuteau, MD, Chief Executive Officer of Axsome. “As highlighted in the FDA’s Patient-Focused Drug Development Initiative report The Voice of the Patient: Fibromyalgia, patients with fibromyalgia experience debilitating widespread pain, fatigue, and functional impairment, and report living with constant stigmatization, anxiety, depression, and fear of ongoing or worsening symptoms. We are excited and motivated by the potential for AXS-14 to address this high unmet medical need for patients, as evidenced by the consistent efficacy demonstrated to date across a broad range of fibromyalgia symptoms, including significant improvements in pain, function, and fatigue, in the completed trials.”

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About AXS-14

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AXS-14 (esreboxetine) is a highly selective and potent norepinephrine reuptake inhibitor for the management of fibromyalgia and other conditions. Esreboxetine, the SS-enantiomer of reboxetine, is more potent and selective than racemic reboxetine. AXS-14 is an investigational drug product not approved by the FDA.

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About Fibromyalgia

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Fibromyalgia is a chronic debilitating disorder characterized by widespread pain, fatigue, disturbed sleep, depression, and cognitive impairment.¹ Other symptoms of this disorder can include tingling in the hands and feet and headaches.¹ Fibromyalgia has considerable detrimental effects on physical, emotional, social, and day-to-day functioning.¹ Fibromyalgia is considered to be mediated mainly in the central nervous system. Approximately 17 million Americans, 90% of whom are women, are estimated to suffer from fibromyalgia.² Treatment options for fibromyalgia are limited with only three pharmacologic treatments currently approved by the FDA.

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About Axsome Therapeutics

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Axsome Therapeutics is a biopharmaceutical company leading a new era in the treatment of central nervous system (CNS) conditions. We deliver scientific breakthroughs by identifying critical gaps in care and develop differentiated products with a focus on novel mechanisms of action that enable meaningful advancements in patient outcomes. Our industry-leading neuroscience portfolio includes FDA-approved treatments for major depressive disorder, excessive daytime sleepiness associated with narcolepsy and obstructive sleep apnea, and migraine, and multiple late-stage development programs addressing a broad range of serious neurological and psychiatric conditions that impact over 150 million people in the United States. Together, we are on a mission to solve some of the brain’s biggest problems so patients and their loved ones can flourish. For more information, please visit us at www.axsome.com and follow us on LinkedIn and X.