On 10 May, Otsuka Pharmaceutical and Lundbeck announced that the US Food and Drug Administration (FDA) has approved the supplemental New Drug Application (sNDA) of brexpiprazole for use in the treatment of agitation associated with dementia due to Alzheimer’s disease (AD). With this approval, brexpiprazole becomes the first drug approved in the US for agitation associated with dementia due to AD. Brexpiprazole was approved in the US in 2015, as an adjunctive therapy to antidepressants in adults with major depressive disorder and as a treatment for schizophrenia in adults. The FDA granted the application based on data from two Phase III clinical trials evaluating the safety and efficacy of brexpiprazole in people with agitation in AD.
The primary endpoint across the studies was improvement from baseline in the Cohen-Mansfield Agitation Inventory (CMAI) total score while the key secondary endpoint was an improvement from baseline to week 12 in the Clinical Global Impression-Severity (CGI-S) score related to agitation. The two randomised, double-blind and placebo-controlled fixed-dose Phase III studies showed a statistically significant treatment effect in the reduction of agitation over a 12-week treatment period. Brexpiprazole demonstrated a 31% improvement from baseline agitation symptoms compared to placebo. In addition, results showed that brexpiprazole was well-tolerated with a safety profile consistent with the known safety profile of brexpiprazole in other indications.