After years of clinical trials and industry anticipation, the FDA has red-lighted Lykos Therapeutics’ midomafetamine (MDMA)-assisted therapy for treating PTSD.
The agency requested that Lykos conduct an additional Phase 3 trial to further study the therapy’s safety and efficacy. This comes after FDA advisors voted down MDMA therapy in June, saying the treatment lacked data about the risks of the therapy and the efficacy.
“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,” Amy Emerson, CEO of Lykos Therapeutics, said in a statement. “While conducting another Phase 3 study would take several years, we still maintain that many of the requests that had been previously discussed with the FDA and raised at the Advisory Committee meeting can be addressed with existing data, post-approval requirements or through reference to the scientific literature.”
Lykos Therapeutics, the commercial spinout of the Multidisciplinary Association for Psychedelic Studies (MAPS), revealed that it would request a meeting with the FDA to ask the agency to reconsider the decision and further discuss its recommendation for resubmission.
MAPS has been researching the use of MDMA for PTSD for years. It landed an FDA breakthrough designation in 2017. Many in the industry pinned their hopes on this research, leading to the first FDA-approved psychedelic therapy.
While this is a significant blow for the burgeoning psychedelics industry, which is hungry for regulatory approval, it’s likely not the end of the road.
“In the big picture, this decision is a bump in the road but not something that will drastically alter the course of psychedelic medicine. The FDA is providing guidance to the industry about what needs to be true for a psychedelic medicine to be approved, and other organizations with drugs in the pipeline will use that guidance to achieve approval of MDMA, psilocybin, LSD, and other drugs in the pipeline,” Michael Petegorsky, chief strategy officer at Mindbloom, told Behavioral Health Business in an email. “This is a difficult learning process that will ultimately lead to the approval of a number of psychedelic medicines. It’s just unfortunate that so many people have to suffer without treatment while this process unfolds.”
Mindbloom provides at-home ketamine services.
Psychedelics — including ketamine, LSD, MDMA and psilocybin — have been lauded as potential treatments for behavioral health conditions including PTSD, alcohol use disorder and depression.
Many have called for making the substances easier to research and study. In fact, in 2023, a bipartisan group of lawmakers introduced an updated version of the Breakthrough Therapies Act to the Senate, which would make it easier to use Schedule 1 drugs with a breakthrough therapy designation. Specifically, it would allow drugs, including MDMA and psilocybin, which have the breakthrough designation, to be transferred from a Schedule I to Schedule II drug, enabling more patient access and more clinical investigation.
“In response to the FDA’s rejection, Lykos has formally requested a reconsideration of the decision,” Petegorsky said. “I think we’ll see real outrage from the public – especially from veterans and members of Congress – which could propel this issue onto the national political stage during the upcoming election cycle. There’s a small but meaningful chance the FDA may reverse its decision in the coming months. In the meantime, ketamine therapy remains the only legal psychedelic medicine. Studies have shown that ketamine can be highly effective for treatment of PTSD, depression, and other mental health conditions, offering hope to people in need of immediate relief.”
Lykos isn’t the only psychedelic company looking to land FDA clearance. Petegorsky noted that Compass Pathways is developing psilocybin therapy for treatment-resistant depression and is currently in stage 3 clinical trials.
