Investigational Drug for Depression Receives Breakthrough Therapy Designation From FDA

February 4, 2016

The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to rapastinel, which comes after the medication received a 2014 Fast Track Designation from the FDA. Rapastinel, an investigational medication for adjunctive treatment of major depressive disorder, is an intravenous formulation of a novel NMDA receptor agonist and is produced by Allergan. 

In a Phase 2 clinical trial of patients with treatment resistant depression, those receiving a single dose of rapastinel experienced antidepressant effects one day after the dose. The Phase 2 studies also indicated that rapastinel has a sustained antidepressant effect, and researchers reported that no psychotomimetic or hallucinogenic side effects were observed. Rapastinel was formerly known as GLYX-13. 

"Nonresponse to antidepressants is a key reason that major depressive disorder is one of the United States' leading public health problems and there is a great need for new therapies that are truly different from those that are currently used," Michael E. Thase, MD, Professor of Psychiatry, Perelman School of Medicine of the University of Pennsylvania, said in a press release. He added that rapastinel operates using a different pathway than traditional antidepressants and might work more quickly than current treatments. 

The Breakthrough Therapy designation is meant to expedite development and review of new medicines that show substantial benefits in preliminary clinical trials over existing treatments for serious or life-threatening diseases. 

Allergan plans to start phase 3 studies of rapastinel in 2016. 

—Lauren LeBano