MDMA treatment for PTSD: FDA rejects new MDMA therapy for PTSD, calls for more trial data

The Food and Drug Administration (FDA) has rejected MDMA which is also known as ecstasy, as a treatment for post-traumatic stress disorder (Post-traumatic stress disorder (PTSD)), drugmaker Lykos Therapeutics said in a news release. MDMA is an entactogen, a class of psychoactive drugs that produce experiences of emotional communion, connection and emotional openness.

According to a CNN report, this is the first time the FDA has considered a psychedelic drug for medical use. The novel treatment has the potential to transform a field with significant needs, but the review process has also raised questions about how it should be used in a clinical setting. MDMA is currently classified as a Schedule I drug under the Controlled Substances Act, or a banned substance that “has no currently accepted medical use and a high potential for abuse.” Reclassifying the drug to a lower tier would be a notable change that would involve multiple federal agencies.

Lykos Therapeutics had asked the FDA to approve the drug as part of a treatment regimen, given alongside talk therapy. The FDA’s decision came after an independent advisory committee in June declined to recommend approval of the drug, saying there was insufficient evidence that the therapy was safe and effective.

The FDA said there were several problems, including poorly designed studies, allegations of sexual misconduct during a mid-stage clinical trial and the potential for serious health risks after taking the drug, including heart problems and abuse. According to an NBC News report, a review by FDA scientists, released before the June meeting, also raised concerns about how the trials were conducted, including that several patients and therapists were likely able to guess who was given the drug and who was given the placebo.

Holly Fernandez Lynch, an assistant professor of medical ethics at the University of Pennsylvania, said: “I think this will be a temporary setback. The advisory committee and the FDA have given very clear directions of what they are looking for in terms of study design and adverse event reporting, so Lykos and other companies should know pretty clearly how to proceed going forward if they want psychedelics approved.”

The FDA has also asked the company to complete a Phase 3 clinical trial to further study the drug’s safety and efficacy. Lykos added that the agency expressed concerns similar to those raised during the advisory committee meeting in June. The drugmaker said it plans to ask the FDA to reconsider the decision.

Lykos CEO Amy Emerson said in a press release: “The FDA’s request for another study is deeply disappointing, not only to all those who dedicated their lives to this pioneering effort, but most importantly to the millions of Americans with PTSD, along with their loved ones, who have not seen any new treatment options in more than two decades.”

In a statement, an FDA spokesperson said the agency recognizes there is a “significant need” for additional treatment options for PTSD. However, “as discussed at the advisory committee meeting, there are significant limitations in the data contained in the application that prevent the agency from concluding that this drug is safe and effective for the proposed indication,” the spokesperson said.