Shares of Axsome Therapeutics (NASDAQ:AXSM) climbed roughly 14% after the U.S. Food and Drug Administration granted approval for AUVELITY to treat agitation associated with dementia due to Alzheimer’s disease. The approval, announced on Thursday, represents the second neuropsychiatric indication for the drug, which already holds approval for major depressive disorder in adults.
The regulatory decision followed an expedited development and review pathway: AUVELITY received FDA Breakthrough Therapy designation during development and was considered under Priority Review. The drug's mechanism involves modulation of the NMDA and sigma-1 receptors through its dextromethorphan component, positioning it as a first-in-class therapy for agitation in Alzheimer’s disease. AUVELITY also contains bupropion, which raises dextromethorphan blood concentrations by inhibiting the cytochrome P450 2D6 enzyme.
Regulatory support for the approval came from two Phase 3 trials. The 5-week ADVANCE-1 study showed that AUVELITY produced a statistically significant improvement in agitation symptoms compared with placebo, measured by the Cohen-Mansfield Agitation Inventory total score at Week 5. The ACCORD-2 trial, a longer-term randomized withdrawal study, found that patients who continued AUVELITY had a statistically significantly longer time to relapse of agitation symptoms than those who were switched to placebo.
Agitation is a common feature of Alzheimer’s disease, appearing in up to 76% of patients and ranging from restlessness and pacing to verbal and physical aggression. The condition affects a broad population, with more than 7 million Americans reported to have Alzheimer’s disease.
Safety findings from the ADVANCE-1 trial identified dizziness and dyspepsia as the most frequently observed adverse reactions. The rate of treatment discontinuation due to adverse events was 1.3%, the same percentage observed in the placebo group. Across clinical trials and real-world use, AUVELITY has been administered to more than 300,000 patients.
Axsome said it will host a webcast on Friday, May 1, at 8:00 AM Eastern Time to discuss the approval and next steps.
Context
This approval marks a new labeled use for AUVELITY within neuropsychiatry, adding to its existing indication for major depressive disorder in adults. The regulatory pathway and supporting Phase 3 results underpin the company's announcement and the market reaction.