Psilocybin and methylone: FDA moves to fast-track review

Some psychedelic drugs, once considered fringe, are now getting a step closer to possibly being approved for use as mental health treatments in the United States.

The US Food and Drug Administration on Friday issued national priority vouchers to three companies studying these psychedelic-based medicines: psilocybin, the active ingredient in “magic mushrooms,” for treatment-resistant depression; psilocybin for major depressive disorder; and methylone, a drug similar to MDMA, for post-traumatic stress disorder – better known as PTSD.

These vouchers were granted under the FDA Commissioner’s National Priority Voucher pilot program, which launched last year as a way to fast-track the review process for certain drugs and biological products seeking approval, potentially cutting down the time it takes for them to reach patients.

The FDA also is now allowing an early phase clinical study of a drug based on the psychedelic ibogaine as a potential treatment for alcohol use disorder to move forward, making the trial the first instance in which the FDA has allowed a clinical study in the United States of the drug, which is derived from the African Tabernanthe iboga plant. While the study has been allowed to proceed, this move does not mean the drug has been approved or found to be safe or effective. The agency says it will continue to review data as it becomes available.

“We are accelerating the research, approval, and responsible access to promising mental health treatments – including psychedelic therapies like ibogaine – to confront our nation’s mental health crisis head-on, especially for our veterans,” Health and Human Services Secretary Robert F. Kennedy, Jr. said in a news release Friday. “The FDA will prioritize therapies with Breakthrough Therapy designation, where early evidence shows meaningful improvement over existing options for serious mental illness.”

The FDA also announced that it plans to release final guidance to provide recommendations to sponsors developing these products.

This latest action by the FDA follows an executive order that President Donald Trump signed Saturday, directing FDA Commissioner Dr. Marty Makary to prioritize psychedelic drugs that have been granted “breakthrough therapy” status – a designation reserved for therapies that show substantial promise over existing treatment options.

Trump’s executive order calls for accelerating both research on and access to psychedelic treatments, including a $50 million investment in state governments to study how psychedelics might benefit people struggling with mental health disorders.

“These medications have the potential to address the nation’s mental health crisis, including conditions like treatment-resistant depression, alcoholism and other serious mental health and substance abuse conditions,” Makary said in a news release Friday. “As this field moves forward, it is critical that their development is grounded in sound science and rigorous clinical evidence. We owe it to our nation’s veterans and all Americans who are suffering from these conditions to evaluate these potential therapies with urgency.”

Psychedelics are a powerful group of drugs that can alter perception, mood and cognition. Some scientists, clinicians and patients have argued that they could offer real hope for treating anxiety, depression, PTSD or other mental illnesses – especially for people whose conditions haven’t improved with traditional treatments. But these drugs aren’t risk-free, and experts caution that their potential downsides need to be taken seriously.

Many psychedelics are currently classified as Schedule I substances by the US Drug Enforcement Administration – the strictest category – reserved for substances it considers to have “no currently accepted medical use and a high potential for abuse.”

But “the FDA has a system with the DEA, by which, when a drug becomes FDA-approved and the FDA sets clear criteria, then it can be given with a transient de-scheduling that is exempt from those enforcement rules, because it’s given in a hospital setting or in a clinic setting under clinical supervision,” Makary told CNN’s Kaitlan Collins on Monday. “If they do get approved, these are not the medications you’ll just pick up at a pharmacy. These are given in a controlled, supervised setting in a hospital.”

The executive order essentially is “to grease the wheels internally within government” to make things move faster for psychedelic medicines seeking FDA approval, said Melissa Lavasani, founder and chief executive of the advocacy group Psychedelic Medicine Coalition, which works to pave the way for safe, effective psychedelic medicines to enter mainstream health care.

“This is significant in that it speeds up access. It speeds up these drugs being available on the market, and it can advance dialogues about what insurance coverage is going to look like and what health care infrastructure needs to be,” Lavasani said this week.

She added that prior to the executive order, some psychedelic companies have tried to get fast-track reviews and were denied.

“There’s not a question of if the Trump administration will allow FDA approvals for psychedelics, it’s ‘when this happens, what do we need to do to be prepared for this?’ So that this can get rolled out properly, so that folks can have access, so that insurance can eventually cover this and we can come up with some training and credentialing and protocols,” Lavasani said. “It’s going to be a trial and error process, and it’s not going to happen overnight. Even with an executive order, this stuff takes a lot of time.”

There has been a growing movement of some scientists, communities and patients advocating for research on the therapeutic effects of psychedelic drugs and broader access, said Dr. David Hellerstein, a research psychiatrist at the New York State Psychiatric Institute and professor at Columbia University Irving Medical Center, who studies the therapeutic potential of psychedelics.

“The psychedelic movement and landscape at this point is a big tent. You have shamans and gurus and people from indigenous communities. You have many, many research groups who are very interested in psychedelics from a scientific point of view. You have the private equity and other finance folks who are interested in commercial development. You have many underground clinicians throughout the country who are providing these drugs, even though they’re officially illegal, to people who want them. You also have military veterans who suffer from PTSD and TBI who want effective treatment,” Hellerstein said.

“What do these people have in common? Not much, except they’re interested in more access,” he said. “It’s this very massive, diverse movement that crosses many boundaries of different interests.”

The American Psychiatric Association said it “welcomes federal investment in research into psychedelic therapies” as potential treatment options for serious mental illnesses, but the organization has some concerns about fast-tracking FDA approvals unless there is enough rigorous clinical trial data to evaluate the risks versus benefits of certain therapies.

“At the same time, current scientific evidence is not sufficient to support use of these therapies outside of approved research settings – which is why the executive order’s emphasis on clinical trials is important. Rigorous study, strong patient protections, and physician-led oversight will be essential to ensure safety and effectiveness,” Dr. Marketa Wills, CEO and medical director of the American Psychiatric Association, said in an email.

“Serious mental illness remains a major public health concern, and patients need treatments they can trust to be both safe and effective. The APA advocates for a rigorous, research-driven approach to psychedelic therapies. Solid evidence and patient safety need to be the priority,” she said, adding that “psychiatrists should play a central role in evaluating and delivering new treatments.”