Axsome Therapeutics, Inc. (NASDAQ: AXSM) has recently 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. The FDA found the NDA to be incomplete for substantive review, specifically citing issues with the second of the two placebo-controlled trials submitted. The second trial's primary endpoint was at 8 weeks and used a flexible-dose paradigm, which the FDA did not consider adequate and well-controlled. However, the first placebo-controlled trial, with a 12-week endpoint and a fixed-dose paradigm, was deemed adequate and well-controlled.
In response to the FDA's feedback, Axsome plans to conduct an additional controlled trial that will use a fixed-dose paradigm and a 12-week primary endpoint, as requested by the FDA. This trial is anticipated to be initiated in the fourth quarter of 2025.
The company's Chief Executive Officer, Dr. Herriot Tabuteau, expressed the company's commitment to addressing the needs of the approximately 17 million patients in the U.S. living with fibromyalgia. Dr. Tabuteau also emphasized the potential of axs-14 to address the high unmet medical need for patients, citing consistent efficacy demonstrated across a broad range of fibromyalgia symptoms in completed trials.
About axs-14, it is described as a highly selective and potent norepinephrine reuptake inhibitor for the management of fibromyalgia and other conditions, and is an investigational drug product not approved by the FDA.
Fibromyalgia is characterized by widespread pain, fatigue, disturbed sleep, depression, and cognitive impairment, with approximately 17 million Americans, 90% of whom are women, estimated to suffer from the condition.
Axsome Therapeutics is a biopharmaceutical company specializing in the treatment of central nervous system (CNS) conditions and has an industry-leading neuroscience portfolio that 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.
The company's mission is to deliver scientific breakthroughs by developing differentiated products with a focus on novel mechanisms of action, aiming to enable meaningful advancements in patient outcomes for over 150 million people in the United States impacted by serious neurological and psychiatric conditions. Today the company's shares have moved 1.13% to a price of $111.91. For the full picture, make sure to review Axsome Therapeutics's 8-K report.
