Axsome’s Therapy for Agitation in Alzheimer’s Granted FDA Fast Track Designation

Magdalena Kegel avatar

by Magdalena Kegel |

Share this article:

Share article via email
AXS-05 for Alzheimer's

Axsome Therapeutics’ investigational drug AXS-05 was recently granted fast track designation by the U.S. Food and Drug Administration (FDA) for the treatment of agitation in patients with Alzheimer’s disease.

The designation intends to speed up the development and regulatory handling of the drug, which is currently in the planning stage for a Phase 2/3 clinical trial.

“Agitation is reported in nearly half of individuals living with Alzheimer’s disease, results in distress to patients and caregivers, and has significant consequences, including early nursing home placement and increased mortality,” Herriot Tabuteau, MD, the CEO of Axsome, said in a press release.

Symptoms come in the form of irritability, aggressive behaviors, emotional distress, and uninhibited behaviors. The symptoms are also linked to lower functional capacity, increasing the burden on caregivers and often leading to earlier placement in a nursing home.

Treatments aimed at specific symptoms that might improve the quality of life of patients are increasingly important, especially with the recent failings in the development of drugs to treat or prevent Alzheimer’s.

“The receipt of Fast Track designation from the FDA highlights the serious nature of this condition, the lack of FDA-approved treatments, and the potential of AXS-05 to address this high unmet medical need,” Tabuteau said.

AXS-05 is a combination of the two drugs bupropion and dextromethorphan. Both have extensive actions on brain neurotransmission. In addition to these effects, bupropion also increases the concentration of dextromethorphan in the body after dosing.

The fast track designation offers several benefits to a drug developer. Improved communications with the FDA, as well as the possibility of submitting a New Drug Application (NDA) on a rolling basis, make the hurdles of drug development and regulatory evaluation less time consuming. Once an NDA is submitted, it is also eligible for priority review by the FDA.

The treatment is currently also being explored in a Phase 3 trial (NCT02741791) as an option for treatment-resistant depression.

While there are no currently approved medications to treat agitation in Alzheimer’s disease, Axsome is not operating alone in the development of such treatments.

Several other companies, including Intra-Cellular Therapies and Alkermes, have candidates in different phases of clinical development. Only a week ago, Otsuka and Lundbeck reported that the schizophrenia medication Rexulti (brexpiprazole) was effective against agitation in Alzheimer’s patients in two completed Phase 3 trials.