FDA declines to approve novel MDMA therapy to treat PTSD, with request for more trial data

The US Food and Drug Administration has declined to approve an application for the use of MDMA, commonly known as ecstasy, as a treatment for post-traumatic stress disorder, according to a news release from drug sponsor Lykos Therapeutics. The company says the agency has requested an “additional Phase 3 trial to further study the safety and efficacy” of the treatment.

The US Food and Drug Administration has declined to approve an application for the use of MDMA, commonly known as ecstasy, as a treatment for post-traumatic stress disorder, according to a news release from drug sponsor Lykos Therapeutics. The company says the agency has requested an “additional Phase 3 trial to further study the safety and efficacy” of the treatment. The initial application from Lykos presented positive data from two late-stage clinical trials that used MDMA in combination with talk therapy to treat PTSD. About 200 people participated in the Lykos trials, with a treatment program that involved three eight-hour sessions in which they took MDMA in the presence of two therapists. The MDMA sessions were spaced about a month apart, along with additional talk therapy sessions. MDMA is an entactogen, a class of psychoactive drugs that produce experiences of emotional communion, relatedness and emotional openness. 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 need, 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 prohibited substance that has “no currently accepted medical use and a high potential for abuse.” Reclassifying the drug to a lower level would be a marked shift involving multiple federal agencies. In June, an independent FDA advisory committee voted overwhelmingly against the treatment, citing concerns about the integrity of the particular trials up for review. When reviewing the trial data from Lykos Therapeutics, many of the committee members applauded the exploration of this line of treatment and lauded the potential it holds but raised critical doubts – notably regarding missing information about safety and bias in efficacy data. They said it was also difficult to parse how much the accompanying talk therapy contributed to outcomes, which is an area that the FDA does not regulate. “Participants appear to experience rapid, clinically meaningful, durable improvement in their PTSD symptoms,” the FDA wrote in a briefing document about the studies before the committee meeting.