Psilocybin for major depression granted Breakthrough Therapy by FDA

The US Food and Drug Administration (FDA) has granted psilocybin therapy a Breakthrough Therapy designation for the second time in a year, this time with a view on accelerating trials testing its efficacy treating major depressive disorder (MDD).

Back in late 2018, the FDA granted Breakthrough Therapy status to the ongoing work from COMPASS Pathways investigating psilocybin, the key psychoactive compound in magic mushrooms, as a therapy for treatment-resistant depression. A large, multi-center Phase 2 trial spanning the US, UK and Europe is currently underway testing a variety of dosing strategies.

This new FDA Breakthrough Therapy approval focuses on a seven-site, Phase 2 trial currently underway in the United States. Coordinated by a non-profit research organization called the Usona Institute, the trial is exploring the antidepressant properties of a single psilocybin dose in treating patients with major depressive disorder.

Last year’s Breakthrough Therapy designation was targeted at the drug’s efficacy for treatment-resistant depression (TRD). That particular clinical classification categorizes patients suffering from MDD who have not responded effectively to at least two different pharmacological antidepressant treatments during a current depressive episode. It is estimated between 10 and 30 percent of MDD patients fall into the category of TRD.

The new FDA approval focuses on Usona’s research into the broader condition of MDD, characterized by episodes of severe depression that last more than two weeks. Hundreds of millions of people around the world suffer from these kinds of acute major depressive episodes every year.

“The results from previous studies clearly demonstrate the remarkable potential for psilocybin as a treatment in MDD patients, which Usona is now seeking to confirm in its own clinical trials,” says Usona’s director of clinical and translational research, Charles Raison. “What is truly groundbreaking is FDA’s rightful acknowledgement that MDD, not just the much smaller treatment-resistant depression population, represents an unmet medical need and that the available data suggest that psilocybin may offer a substantial clinical improvement over existing therapies.”