Dexmedetomidine Oral Film Receives Breakthrough Tx Status For Acute Arousal With Dementia

The Food and Drug Administration (FDA) has granted BXCL501 (dexmedetomidine; BioXcel Therapeutics) the Breakthrough Therapy designation for the acute treatment of arousal associated with dementia.

Dexmedetomidine is currently available as a solution for intravenous infusion for use in clinical anesthesia and sedation in an intensive care unit. BXCL501 is an oral thin-film formulation of dexmedetomidine, a selective alpha-2a receptor agonist.

The designation is supported by data from the randomized, double-blind, placebo-controlled Phase 1b / 2 TRANQUILITY study ( NCT04251910), which assessed the efficacy, safety, and pharmacokinetics of BXCL501 in 54 patients 65 years of age and older related to dementia; 87% of the study participants had Alzheimer’s. Patients were randomly assigned to receive 30 mcg (n = 16), 60 mcg (n = 20), 90 mcg (n = 4), or placebo (n = 14) BXCL501.

The results showed a statistically significant reduction in agitation measures (positive and negative syndrome scale arousal component; Pittsburgh agitation scale and modified Cohen-Mansfield agitation inventory) 2 hours after the dose with the 30 mcg and 60 mcg doses compared to placebo. The most common adverse event reported was mild or moderate sleepiness.

“Treating dementia, especially in the elderly, is a significant challenge for doctors and nurses as there are currently no FDA-approved therapies and black box warnings are attached to off-label drugs,” said Vimal Mehta , Chief Executive Officer of BioXcel. “The FDA’s decision to grant Breakthrough Therapy further underscores the significant unmet need for a new treatment for this underserved patient population and underscores the potential of BXCL501 to become the first therapeutic option if approved to address it address debilitating medical condition. ”

The company recently filed a new drug application for BXCL501 for the treatment of agitation related to schizophrenia and bipolar disorder I and II.


  1. BioXcel Therapeutics Receives FDA Breakthrough Therapy Designation for BXCL501 for the acute treatment of arousal associated with dementia. [press release]. New Haven, CT: BioXcel Therapeutics, Inc .; March 15, 2021.
  2. BioXcel Therapeutics announces that BXCL501 has met the primary and all secondary endpoints in the TRANQUILITY Phase 1b / 2 study for the acute treatment of agitation in dementia, including Alzheimer’s disease. [press release]. New Haven, CT: BioXcel Therapeutics, Inc .; January 5, 2021.

This article originally appeared on MPR


Alzheimer’s Disease and Dementia Dementia Treatments

Related Articles