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Axsome Therapeutics Receives FDA Breakthrough Therapy Designation for AXS-12 for the Treatment of Narcolepsy
Designation offers potential for expedited development and review FDA Orphan Drug Designation previously granted to Axsome for AXS-12 in narcolepsy Third FDA
About this update from Axsome Therapeutics, Inc.
[{"type":"text","content":"Designation offers potential for expedited development and review\n FDA Orphan Drug Designation previously granted to Axsome for AXS-12 in narcolepsy Third FDA Breakthrough Therapy designation received by Axsome NEW YORK, Aug. 05, 2020 (GLOBE NEWSWIRE) -- Axsome Therapeutics, Inc. (NASDAQ: AXSM), a 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-12 for the treatment of cataplexy in patients with narcolepsy. AXS-12 is a novel, oral, highly selective and potent norepinephrine reuptake inhibitor. Axsome previously received Orphan Drug Designation from the FDA for AXS-12 for the treatment of narcolepsy. Narcolepsy is a debilitating, neurological condition characterized by excessive daytime sleepiness (EDS) and cataplexy, a sudden loss of muscle tone triggered by strong emotions. 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 Breakthrough Therapy designation for AXS-12 for the treatment of cataplexy in narcolepsy was supported by the positive results from the Phase 2 CONCERT study, a randomized, double-blind, placebo-controlled, crossover, multicenter U.S. trial. In the trial, 21 patients with a diagnosis of narcolepsy with cataplexy were treated for 2 weeks with AXS-12 or with placebo, followed by a crossover to the other treatment after a 1-week down-titration and washout period. AXS-12 met the primary endpoint demonstrating a highly statistically significant reduction from baseline in the mean weekly number of cataplexy attacks, averaged for the 2-week treatment period (overall treatment effect), as compared to placebo (p","length":2428,"tagName":"div"}]