FDA Nixes MDMA Therapy for PTSD, Shocking Mental Health Advocates

On August 9, 2024, Lykos Therapeutics announced that the Food and Drug Administration (FDA) had declined its application for MDMA-assisted therapy aimed at treating post-traumatic stress disorder (PTSD). This marks the FDA’s first rejection of a psychedelic drug application, disappointing many advocates for psychedelic therapies.

Investors and researchers had anticipated a boom in psychedelic treatments, with MDMA being the initial candidate in a growing pipeline. However, following the FDA’s rejection, stock prices across the psychedelic sector plummeted. The FDA did provide feedback on how Lykos could potentially refine its application for future approval.

Days after Lykos’ announcement, the journal Psychopharmacology retracted three studies on MDMA-assisted therapy due to ethical concerns, raising further questions about the integrity of the research conducted.

In an interview with The Conversation, Benjamin Y. Fong, a drug researcher at Arizona State University, discussed the implications of the FDA’s decision for the future of psychedelic medications. MDMA, commonly known as “molly” or “ecstasy,” is an amphetamine often associated with recreational use for its euphoric effects. Despite being classified as a Schedule I drug, indicating no accepted medical use, recent clinical trials have suggested its potential benefits in treating PTSD when combined with psychotherapy.

PTSD affects an estimated 9 to 13 million adults in the U.S. annually, and the landscape of treatment has not significantly evolved in over two decades. Lykos Therapeutics submitted its application in February 2024, proposing a form of therapy designed to help patients tap into their “inner healing intelligence.”

The FDA’s advisory committee overwhelmingly opposed Lykos’ claims of efficacy, citing numerous issues including “functional unblinding,” where participants could discern whether they received a psychoactive substance, thus skewing results. Critics questioned the psychotherapy approach used alongside MDMA and ethical concerns related to reported misconduct during trials.

The FDA, while not obligated to follow the advisory panel’s recommendations, usually adheres to their insights. As such, the decision was not entirely unexpected.

Moving forward, the FDA has mandated an additional phase 3 study to rigorously confirm the treatment’s safety compared to traditional therapies. Lykos intends to seek reconsideration of the FDA’s ruling while addressing the analysis and guidance provided.

Lykos CEO Amy Emerson expressed profound disappointment and indicated that the proposed new trial would likely span several years. The retraction of prior research only complicates the path ahead.

Other companies in the psychedelic space, such as Compass Pathways, are currently preparing their applications for FDA approval with differing methodologies. Compass is advancing with phase 3 trials for synthetic psilocybin, suggesting a distinction in trial design that may adhere more closely to FDA expectations.

The unique challenges of psychedelic drug development extend beyond Lykos’ setbacks. The nature of psychedelics, which can induce intense alterations of consciousness, often leads to ethical scrutiny and difficulties in maintaining research integrity. While the recent FDA ruling pertains significantly to Lykos’ specific application, it sheds light on broader concerns faced by emerging psychedelic therapies in the clinical arena.