The U.S. Food and Drug Administration (FDA) just made a decision that could have large implications for the future of the 13 million Americans living with post-traumatic stress disorder (PTSD) each year.
The FDA on Aug. 9 decided not to approve a new drug application from the public benefit company Lykos Therapeutics for its MDMA – also known as molly or ecstasy — capsules to be used, alongside therapy, to treat PTSD.
Lykos said on Friday that it had received a complete response letter from the agency regarding its application. The company said that the FDA had completed its review of of the application and determined it could not approve it based on currently submitted data.
Instead, the FDA asked Lykos to conduct an additional phase 3 trial to further study the safety and efficacy of the treatment.
“The FDA request for another study is deeply disappointing, not just for all those who dedicated their lives to this pioneering effort, but principally for the millions of Americans with PTSD, along with their loved ones, who have not seen any new treatment options in over two decades,” said Lykos CEO Amy Emerson in a statement.
The company now plans to request a meeting with the FDA to request a reconsideration of the decision.
Lykos, a spin-off of the non-profit Multidisciplinary Association for Psychedelic Studies (MAPS), submitted its application for the experimental treatment late last year. The FDA fast-tracked the application, granting it priority review in February and gave itself the August deadline to make a decision on whether to approve the first-of-its-kind treatment.
Lykos had outlined how its MDMA-assisted therapy for PTSD works in two phase 3 clinical trials.
In the studies, patients were given MDMA capsules made by Lykos along with talk therapy in three eight-hour sessions, overseen by two specially-trained therapists. The sessions were spread about four weeks apart.
According to results from the the most recent trial, about 71% of participants who received MDMA-assisted therapy no longer met the criteria for PTSD after 18 weeks, compared with 48% of the patients who received a placebo.
However, the company’s application first hit a major setback in June when a FDA advisory committee recommended that the health regulator not approve the treatment. It was the first FDA advisory committee review of a potential new PTSD treatment in 25 years.
The committee ultimately took issue with the data provided by Lykos and its clinical trial processes. Because MDMA produces alterations in mood and cognition, patients may know if they received the treatment or a placebo, making it nearly impossible to carry out a double-blind study— the gold standard for clinical trials.
Quartz talked to two experts in the field of psychedelic treatments — before the FDA’s decision came in — to get their perspectives on the FDA’s review and what it could mean for the future of psychedelic drugs.
Dr. Gül Dölen, professor of neuroscience and psychology at UC Berkeley
Dr. Gül Dölen has been studying learning, memory, and social behavior for the past 30 years. And in the most recent decade, she has been researching psychedelics and their therapeutic effects.
Her lab found evidence that MDMA and psychedelics in general can open critical periods for social behaviors. Dölen defines critical periods as “windows of time where the brain is especially sensitive to the world around it and can learn from that environment.”
In her view, this is why MDMA, in combination with psychotherapy, has been found to be so effective at treating PTSD.
On Friday, Dölen’s worst case scenario regarding the FDA’s decision came to pass when the agency said Lykos would need to conduct an additional phase 3 trial before its application could be approved.
“That would be massively expensive and take a long time and would probably be, in my view, the most, extremely negative response that they’re likely to have,” said Dölen.
She had hoped that the agency would have instead approved Lykos’s application and requested a phase 4 trial that would have monitored for concerns brought up by the advisory committee such as risks of abuse and cardiotoxicity.
Dölen feared that the FDA, by not approving the treatment, would be signaling to drug companies that psychedelics combined with therapy is not “a viable path forward.” This could result in drug companies doing away with therapy requirements for future drugs.
Mark Rus, CEO of Delix Therapeutics
Mark Rus is the CEO of Delix Therapeutics, a company that is developing psychedelics that have been stripped of their psychoactive properties in order to be able to scale their adoption to a large patient population.
Because these drugs have been found to help repair damaged neurons, Rus hopes they can help treat mental disorders like depression and PTSD, with or without therapy. In addition, he said they could one day help with other neurological conditions, such as Parkinson’s and Alzheimer’s.
This summer, the Department of Defense awarded Delix a grant to advance research into a drug for hearing loss.
Rus predicted that the FDA would possibly delay its decision or ask for more clinical trials.
“While speed to patient is important, it’s also important to do so in a way that is safe and scientifically sound,” said Rus. “And I have no doubt that Lykos will do what they need to do to. It’s not a matter of if but when their treatment will be approved.”
He said that in the short term investors and traders may have a big reaction to the FDA’s decision, but he wouldn’t advise anyone to read too much into this single decision, regardless of the outcome, in regards to the future of psychedelic treatments.
“When you’re in a 20 year drug development life cycle, you think in much more macro terms,” Rus said. “I think the science is incredibly sound. The data is promising. There’s a lot here that will change mental health treatment for the better.”