Axsome Therapeutics, Inc., a clinical-stage biopharmaceutical company developing novel therapies for the management of central nervous system (CNS) disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for AXS-05 for the treatment of major depressive disorder (MDD). AXS-05 is a novel, oral, investigational NMDA receptor antagonist with multimodal activity.
A Breakthrough Therapy designation is granted to potentially expedite development and review timelines for a promising investigational medicine when preliminary clinical evidence indicates it may demonstrate substantial improvement on one or more clinically significant endpoints over available therapies for a serious or life-threatening condition. The designation for AXS-05 in MDD was supported by the recent positive results from the Phase 2 ASCEND study, a randomized, double-blind, active-controlled, multicenter, U.S. trial, in which 80 patients with confirmed moderate to severe MDD were treated with AXS-05 or the active comparator bupropion. In this trial, treatment with AXS-05 resulted in a substantial, rapid, and statistically significant reduction in depressive symptoms as compared to the active comparator bupropion. On the pre-specified primary endpoint, AXS-05 demonstrated a statistically significant average mean reduction from baseline in the Montgomery-Åsberg Depression Rating Scale (MADRS) total score over the 6-week treatment period of 13.7 points for AXS-05 compared to 8.8 for bupropion (p<0.001). AXS-05 was safe and well tolerated with the most commonly reported adverse events in the AXS-05 arm being nausea, dizziness, dry mouth, decreased appetite, and anxiety.
"This Breakthrough Therapy designation from the FDA for AXS-05 in major depressive disorder exemplifies Axsome’s commitment to developing novel medicines that have the potential to significantly improve the lives of patients living with serious CNS disorders," said Herriot Tabuteau, MD, Chief Executive Officer of Axsome. "There is a significant unmet medical need for new and mechanistically differentiated treatments for depression. We look forward to working closely with the FDA over the coming months to expedite the development of AXS-05 for the treatment of major depressive disorder."
About FDA Breakthrough Therapy Designation
Breakthrough Therapy designation is granted by the FDA in order to expedite the development and review of drugs for serious or life-threatening conditions. In order to receive Breakthrough Therapy designation, a drug must demonstrate preliminary clinical evidence that the drug may have substantial improvement on at least one clinically significant endpoint over available therapy. Breakthrough Therapy designation provides an organizational commitment involving senior managers from the FDA, more intensive FDA guidance on an efficient drug development program, and greater access to and more frequent communication with the FDA throughout the entire drug development and review process. It also provides the opportunity to submit sections of a New Drug Application (NDA) on a rolling basis, where the FDA may review portions of the NDA as they are received instead of waiting for the entire NDA submission. In addition, Breakthrough Therapy designated products are eligible for Priority Review, where the FDA has a goal to take action on an application within six months, as opposed to ten months under standard review. Breakthrough Therapy designation does not change the standards for approval.
About Major Depressive Disorder (MDD)
Major depressive disorder (MDD) is a debilitating, chronic, biologically-based disorder characterized by low mood, inability to feel pleasure, feelings of guilt and worthlessness, low energy, and other emotional and physical symptoms, and which impairs social, occupational, educational, or other important functioning. In severe cases, MDD can result in suicide. According to the National Institutes of Health, an estimated 6.7% of U.S. adults, or approximately 16 million, experience MDD each year1. According to the World Health Organization (WHO), depression is the leading cause of disability worldwide, and is a major contributor to the overall global burden of disease2. Nearly two-thirds of diagnosed and treated patients do not experience adequate treatment response with currently ...
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