FDA Declines To Approve MDMA-Assisted Psychotherapy As a PTSD Treatment

The Food and Drug Administration (FDA) declined on Friday to approve MDMA-assisted psychotherapy as a treatment for post-traumatic stress disorder (PTSD). The agency has asked Lykos Therapeutics, which submitted the new drug application (NDA) in February, to conduct an additional Phase 3 clinical trial, which would take years and millions of dollars. It is a major setback that dismayed veteran advocates and others who see MDMA as a potentially life-changing psychotherapeutic catalyst for people struggling with longstanding psychological challenges that currently approved treatments have not adequately addressed.

“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 Amy Emerson, CEO of Lykos Therapeutics, a company created by the Multidisciplinary Association for Psychedelic Studies (MAPS), in a press release. “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.”

The decision was a huge disappointment to advocates of a treatment that the FDA recognized as a “breakthrough therapy” in 2017—especially since the two Phase 3 trials, which were published by Nature Medicine in 2021 and 2023, had found that subjects who took MDMA fared substantially better than subjects who underwent the same psychotherapy but took a placebo. In the 2021 study, two-thirds of the MDMA group “no longer met the diagnostic criteria for PTSD,” compared to a third of the control group. In the 2023 study, more than 70 percent of the MDMA group no longer qualified for a PTSD diagnosis, compared to 48 percent of the control group.

The FDA’s decision nevertheless was unsurprising in light of an advisory panel’s June 4 recommendation against approval. The committee, which included just one scientist with experience in psychedelic research, overwhelmingly concluded that MDMA’s effectiveness had not been adequately established and that its benefits had not been shown to outweigh its risks. The FDA’s concerns are rather puzzling, given that Lykos worked with the agency every step of the way to develop an acceptable research protocol.

The FDA declined to release the letter to Lykos in which it detailed those concerns, which it said is confidential. But the company’s response suggests that the issues identified by the agency are similar to the ones highlighted by its advisers.

The company noted concerns that “Lykos’ clinical data were insufficient to demonstrate durability.” Lykos “believes that the data included in the NDA provide sufficient evidence of efficacy and durability in line with the relevant FDA guidance,” the company said. “FDA’s draft guidance for industry on psychedelic drugs indicates that endpoint data should be collected at 12 weeks; Lykos’ Phase 3 studies collected endpoint data at 18 weeks, with additional exploratory endpoints collected six months or more later.”

The advisory panel also raised “questions about expectancy bias stemming primarily from participants with prior MDMA use.” The concern was that subjects whose previous experience with MDMA was positive would be primed to expect good results in the clinical trials, which might have skewed the results. But Lykos notes that it “aligned with the FDA” on “a variety of bias minimization measures in the st

Read More

Article from Reason.com

Comments

comments