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The US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to psilocybin-assisted therapy from Compass Pathways for patients with treatment-resistant depression (TRD), the manufacturer reports.

This lays the groundwork for an upcoming multicountry study that will assess the safety and efficacy of psilocybin, the psychoactive ingredient in psychedelic or “magic” mushrooms.

Robin Carhart-Harris, Ph.D., head of the psychedelic research group, Imperial College London, United Kingdom, noted in a press release that he and a group of investigators “found promising signals of efficacy and safety as treatment” for refractory depression in their 2015 study.

The new Breakthrough Therapy designation “is a strong endorsement for the potential of psilocybin therapy. We look forward to learning more as further clinical trials are carried out, by our team at Imperial College as well as in Compass’ multi-center trial,” Carhart-Harris said.

Previous research has shown benefit from psilocybin in treating end-of-life anxiety and depression, alcohol and tobacco addiction, and even obsessive-compulsive disorder.

As reported by Medscape Medical News, investigators from Imperial College assessed pre- and posttreatment fMRI data for 19 patients with TRD who received psilocybin in a study published October 2017 in Scientific Reports.

The patients experienced significant improvements in depressive symptoms 1 day posttreatment — and almost half met criteria for a full response at 5 weeks. In addition, whole-brain analyses showed decreases in cerebral blood flow in the temporal cortex and increased resting-state functional connectivity after treatment.

Also commenting on the study at the time for Medscape Medical News, Frederick Barrett, PhD, Behavioral Pharmacology Research Unit, Johns Hopkins School of Medicine, Baltimore, Maryland, called the findings “groundbreaking.”

He added that the study “begins to put some dimensions to the potential neurobiological basis of the therapeutic effects of psychedelics.” However, he cautioned that more research was needed before clinicians should move forward in treating TRD with the drug.

Major Milestone

The FDA designates a drug as a Breakthrough Therapy “if preliminary clinical evidence shows that it may demonstrate substantial improvement over available therapy,” Compass noted in its release. It also highlights “the importance of supporting early research that can be translated to clinically meaningful outcomes.”

Overall, the designation “is a significant milestone for psilocybin therapy and…a testament to work done over many years by research teams in the US, the UK, and Switzerland,” the company added.

It reported that it is now running the first large-scale clinical trial for treating TRD with the drug. The study will be conducted in Europe and North America and will run for approximately 1 year.

David E. Nichols, Ph.D., chairman of the board and co-founder of Heffter Research Institute, Santa Fe, New Mexico, noted that his center “has been helping to design, review, and fund the early phase clinical studies on psilocybin at research institutions in the US and Europe” since 1993.

“We are delighted that psilocybin is being recognized as a Breakthrough Therapy and look forward to continuing our work with researchers and partners around the world so that we can alleviate the suffering caused by mental illness,” Nichols said.

“This is great news for patients,” George Goldsmith, executive chairman at Compass Pathways, added in the same release.

“The FDA will be working closely with us to expedite the development process and increase the chances of getting this treatment to people suffering with depression as quickly as possible,” Goldsmith reported.

Follow Deborah Brauser on Twitter: @MedscapeDeb.

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