FDA Approves First Drug to Treat Alzheimer's Agitation

Megan Brooks

May 11, 2023

The US Food and Drug Administration (FDA) has approved the antipsychotic brexpiprazole (Rexulti, Otsuka and Lundbeck) for agitation associated with Alzheimer's disease (AD), making it the first FDA-approved drug for this indication.

"Agitation is one of the most common and challenging aspects of care among patients with dementia due to Alzheimer's disease," Tiffany Farchione, MD, director of the Division of Psychiatry in the FDA's Center for Drug Evaluation and Research, said in a news release.

Agitation can include symptoms that range from pacing or restlessness to verbal and physical aggression. "These symptoms are leading causes of assisted living or nursing home placement and have been associated with accelerated disease progression," Farchione said.

Brexpiprazole was approved by the FDA in 2015 as an adjunctive therapy to antidepressants for adults with major depressive disorder and for adults with schizophrenia.

Approval of the supplemental application for brexpiprazole for agitation associated with AD dementia was based on results of two randomized, double-blind, placebo-controlled studies.

In both studies, patients who received 2 mg or 3 mg of brexpiprazole showed statistically significant and clinically meaningful improvements in agitation symptoms, as as shown by total Cohen-Mansfield Agitation Inventory (CMAI) score, compared to patients who received placebo.

The recommended starting dosage for the treatment of agitation associated with AD dementia is 0.5 mg once daily on days 1 to 7; it was increased to 1 mg once daily on days 8 through 14 and then to the recommended target dose of 2 mg once daily.

The dosage can be increased to the maximum recommended daily dosage of 3 mg once daily after at least 14 days, depending on clinical response and tolerability.

The most common side effects of brexpiprazole in patients with agitation associated with AD dementia include headache, dizziness, urinary tract infection, nasopharyngitis, and sleep disturbances.

The drug includes a boxed warning for medications in this class that elderly patients with dementia-related psychosis treated with antipsychotic drugs are at an increased risk of death.

The supplemental application for brexpiprazole for agitation had fast-track designation.

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