FDA Advisory Panel Rejects MDMA-Assisted Therapy for PTSD
The panelists expressed concerns about clinical trial data and concluded that the benefits do not outweigh the risks, but the FDA does not have to follow their recommendation.
An independent advisory panel for the FDA rejected the use of MDMA-assisted therapy for post-traumatic stress disorder (PTSD), expressing concern about the reliability of clinical trial data and concluding that the benefits do not outweigh the risks.1 Importantly, the FDA does not have to follow the recommendation of the committee.
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The NDA submission is based on results from the phase 3 MAPP1 and MAPP2 studies. Participants were randomly assigned 1:1 to receive either MDMA or the placebo, in addition to 3 preparatory and 9 integrative therapy sessions. The study authors found that MDMA led to a significant improvement in Clinician-Administered PTSD Scale for DSM-5 score compared with placebo (P<0.0001, d=0.91) and significant decreases in the Sheehan Disability Scale total score (P=0.00116, d=0.43).1
Neither study reported serious adverse events in the MDMA arms.1
The FDA accepted the new drug application (NDA) for midomafetamine capsules (MDMA; Lykos Therapeutics) in February. According to the NDA, the drug would be used in combination with psychological interventions such as psychotherapy and other supportive services for individuals with PTSD. The application was granted priority review.2
According to reporting by the New York Times, panelists questioned the designs of 2 studies submitted by Lykos Therapeutics, particularly the fact that trial participants were largely able to correctly guess whether they had received MDMA or the placebo. Additionally, panelists expressed concern about potential cardiovascular effects of the drug and potential bias among therapists and facilitators involved with study participants.1
Notably, panelists also said they were very concerned about the lack of data from study participants regarding the potential for abuse of the drug.1
"While the recent FDA report raises valid concerns about the use of MDMA, including potential abuse and side effects, it's crucial to recognize the need for diversity in therapeutic approaches to mental health," said Joe Perekupka, CEO of Freespira, an FDA-cleared at-home treatment for PTSD symptoms, in a news release.3
Research into psychedelics presents many logistical challenges, including regulatory hurdles, storing and handling requirements, and record-keeping. In an interview with Pharmacy Times, Mark Bleackley, PhD, chief scientific officer at Incannex Healthcare Limited in Melbourne, Australia, said patient subjectivity also presents a challenge because patient-reported outcomes are the most common barometer for measuring psychedelics' efficacy in mental health.4
Some experts have called for the FDA to reassess its clinical research requirements for psychedelic treatments, potentially allowing a new approach and an adapted framework better suited to these treatments. However, Lykos Therapeutics' MDMA-assisted therapy will continue to undergo FDA evaluation in the traditional framework.
Updated June 5, 2024 at 9 a.m.
