Axsome Therapeutics Updates NDA for AXS-14 Fibromyalgia Treatment

Axsome Therapeutics, Inc., a prominent biopharmaceutical company based in New York, recently disclosed that it has received a Refusal to File (RTF) letter from the U.S. Food and Drug Administration (FDA) regarding its New Drug Application (NDA) for AXS-14 (esreboxetine). This application was intended for the treatment of fibromyalgia, a condition characterized by widespread pain and fatigue. The FDA's decision was based on an initial assessment which found the submission lacking in several significant areas, preventing a full review.

The FDA highlighted that one of the two placebo-controlled trials included in the NDA did not meet the required standards. Specifically, this trial was criticized for having an 8-week primary endpoint and employing a flexible-dose approach. In contrast, the first trial in the submission, which utilized a 12-week primary endpoint and fixed dosing, was deemed satisfactory by the FDA. Importantly, the agency did not question the positive outcomes of the studies, as both trials achieved their primary objectives.

In response to the FDA's feedback, Axsome is planning to conduct an additional controlled trial. This new study will align with the FDA's requirements by using a fixed-dose regimen and a 12-week primary endpoint. The company anticipates launching this trial by the end of 2025. Axsome’s CEO, Herriot Tabuteau, MD, expressed appreciation for the detailed guidance from the FDA, which enables the continued development of AXS-14. Approximately 17 million people in the U.S., predominantly women, are affected by fibromyalgia. The company is eager to proceed with the development of this potential treatment to address the high unmet needs associated with fibromyalgia, a condition with limited treatment options.

Fibromyalgia is a chronic illness that significantly impacts daily life, causing not only physical pain but also emotional and cognitive difficulties. Patients often experience a range of symptoms including fatigue, disturbed sleep, depression, and cognitive impairment. This disorder is believed to primarily affect the central nervous system. Despite the prevalence of fibromyalgia, treatment options remain scarce, with only three pharmacological treatments currently approved by the FDA.

AXS-14, classified as an investigational drug, represents a new approach to managing fibromyalgia. It is a norepinephrine reuptake inhibitor that is both highly selective and potent. AXS-14 is distinguished from racemic reboxetine by its increased potency and selectivity. While promising, it has yet to receive FDA approval.

Axsome Therapeutics is at the forefront of developing innovative treatments for central nervous system disorders. The company focuses on addressing significant gaps in medical care by creating products with unique mechanisms of action that promise meaningful improvements in patient outcomes. Their portfolio includes FDA-approved treatments for conditions such as major depressive disorder, migraine, and excessive daytime sleepiness linked to narcolepsy and obstructive sleep apnea.

In conclusion, while Axsome faces a temporary setback with the RTF letter from the FDA, the company remains committed to advancing AXS-14 through an additional trial that adheres to regulatory stipulations. By addressing the FDA's concerns, Axsome aims to provide a novel therapeutic option for fibromyalgia sufferers, a goal that aligns with their broader mission of tackling pressing neurological and psychiatric challenges.