FDA decision on AXS-05 for Alzheimer’s agitation expected April 30
Agency grants priority review to Axsome Therapeutics' oral therapy
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The U.S. Food and Drug Administration (FDA) granted priority review to an application from Axsome Therapeutics seeking approval of its oral therapy AXS-05 to treat agitation related to Alzheimer’s disease, and expects to decide by April 30, the company said.
Priority review shortens the FDA’s review time to six months from the usual 10 months.
“We are very pleased the FDA has accepted and granted priority review to our supplemental [new drug application] for AXS-05 for the treatment of Alzheimer’s disease agitation,” Herriot Tabuteau, MD, CEO of Axsome, said in a company press release.
Alzheimer’s is a neurological disorder marked by problems with memory and emotional regulation. Agitation is a common Alzheimer’s symptom that can cause substantial distress for patients and their families. AXS-05 contains a combination of two molecules, dextromethorphan and bupropion, both of which are expected to regulate the activity of brain signaling molecules in a way that’s likely to ease agitation.
“Up to 76% of people with Alzheimer’s disease experience agitation, representing a significant unmet medical need for patients and their caregivers, and currently there is a dearth of approved treatments. We look forward to continuing to work with the FDA for the remainder of the review,” Tabuteau said.
Data from trials show promise
Axsome’s application includes data from four Phase 3 clinical trials: ACCORD (NCT04797715), ACCORD-2 (NCT04947553), ADVANCE-1 (NCT03226522), and ADVANCE-2 (NCT05557409).
In all these trials, agitation was measured with a standardized assessment called the Cohen-Mansfield Agitation Inventory (CMAI).
In the ACCORD and ACCORD-2 studies, all participants initially received AXS-05, and those who experienced a reduction in CMAI scores were randomly assigned to either continue the treatment or switch to a placebo. Both trials hit their main goals, showing that patients who switched to the placebo were more likely to have a recurrence of agitation than those who stayed on AXS-05.
Participants in the ADVANCE-1 and ADVANCE-2 studies were randomly assigned to receive AXS-05 or a placebo at the start of the study, with the aim of demonstrating that the therapy was more effective in reducing CMAI scores. The smaller ADVANCE-1 study hit this goal, while the larger ADVANCE-2 study didn’t.
In the larger study, CMAI scores tended to improve with AXS-05 relative to the placebo, but the difference wasn’t statistically significant — meaning it’s mathematically plausible that the difference is due to random chance.
Axsome’s application also includes data from an extension study (NCT06736509) that monitored long-term safety outcomes from patients who had participated in some of the Phase 3 trials.
