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FDA Advisory Committee Rejects First MDMA Treatment for PTSD
The US Food and Drug Administration’s (FDA) Psychopharmacologic Drugs Advisory Committee (PDAC) voted against a midomafetamine (MDMA)-based therapy for the treatment of post-traumatic stress disorder (PTSD) in adults.
The PDAC discussed Lykos Therapeutics' new drug application for MDMA capsules used in combination with psychological intervention for PTSD treatment. MDMA targets multiple neurotransmitters in the brain, including serotonin, noradrenaline, and dopamine.
Regarding whether the available data showed that MDMA is effective in treating patients with PTSD, PDAC voted 2 yes to 9 no. On the question of whether the benefits of MDMA with the FDA’s proposed Risk Evaluation Mitigation Strategy (REMS) outweigh its risks for the treatment of patients with PTSD, the panel voted 1 yes to 10 no.
PDAC's review included the results from two randomized, double-blind, placebo-controlled Phase 3 studies (MAPP1 and MAPP2). These studies assessed the efficacy and safety of MDMA-assisted therapy, which involves using MDMA in combination with psychological intervention. The studied psychological intervention included psychotherapy and other supportive services provided by a qualified health care provider. Both studies reviewed by the PDAC successfully met their primary and secondary endpoints, and their findings were published in Nature Medicine.
Still, the PDAC raised concerns over the data presented in those studies. "It seems like there are so many problems with the data…each one alone might be okay, but when you pile them on top of each other, there's just a lot of questions still I would have about how effective the treatment is and how durable it is," said Melissa Barone, PsyD, a panel member who voted against the treatment's benefit. She went on to say that there wasn’t enough data on diverse populations, that the way the MDMA-assisted therapy was administered was not standardized, and that the psychotherapy involved was “really vague.”
Related>>MDMA-Assisted Therapy Shows Safety and Efficacy for PTSD Treatment
In a May 2024 evidence report, the Institute for Clinical and Economic Review (ICER) also raised concerns about the data supporting the treatment. The ICER report stated that the current publicly available evidence for MDMA-assisted psychotherapy (MDMA-AP) compared to trauma-focused psychotherapies is "insufficient" due to the inability to assess the frequency of misreporting of benefits and/or harms.
That report also stated that, while the study results suggest that MDMA-AP could be beneficial for treating PTSD, there are concerns about the validity of the results due to the unblinded nature of the trials and the strong beliefs of those involved in the trials—investigators, therapists, and patients—about the benefits of MDMA-AP.
In February 2024, the FDA accepted the company’s new drug application and granted the application priority review. The PDAC provides independent recommendations to the FDA, but the FDA is not bound to the committee’s decision. The FDA's approval decision has a target action date of August 11, 2024. If the FDA approves the treatment, the DEA would be required to reschedule MDMA, allowing it to be prescribed for medical use.
"We remain committed to working with the FDA to address outstanding questions so that we may find a path forward to ensure the responsible and careful introduction of MDMA-assisted therapy into the health care system if approved,” said Lykos Therapeutics’ CEO Amy Emerson in a news release.
References
Meeting of the Psychopharmacologic Drugs Advisory Committee (PDAC. June 4, 2024. Accessed June 6, 2024. Videocast available at: https://www.youtube.com/watch?v=JqQKP8gcY1E
