FDA Panel Rejects Lykos MDMA PTSD Drug

An FDA advisory panel recently voted against approving Lykos Therapeutics' midomafetamine (MDMA) capsules for treating post-traumatic stress disorder (PTSD) when used in conjunction with psychological intervention. This decision was based on concerns regarding both efficacy and the risk-benefit profile of the treatment.

The Psychopharmacologic Drugs Advisory Committee (PDAC) was asked to evaluate whether the available data demonstrated MDMA's effectiveness for PTSD patients. In response, the committee members overwhelmingly voted against the treatment, with a tally of 2-9. Subsequently, the panel voted 1-10 against the proposition that the benefits of MDMA, even with the FDA's proposed risk evaluation and mitigation strategy (REMS), outweighed its associated risks for PTSD treatment.

The panel’s review considered the results from two phase 3 clinical trials, known as MAPP1 and MAPP2. These trials were randomized, double-blind, and placebo-controlled, designed to assess the efficacy and safety of MDMA combined with psychological intervention compared to a placebo with psychological intervention in participants suffering from severe or moderate to severe PTSD. Both studies achieved their primary and secondary endpoints, indicating positive outcomes.

However, prior to the advisory committee meeting, significant concerns were raised about these trials. In March, the Institute for Clinical and Economic Review (ICER), a nonprofit organization, published a report criticizing the data from Lykos Therapeutics. The ICER report pointed out issues such as placebo bias and ethical concerns. Specifically, ICER mentioned that some therapists involved in the trials may have influenced patient reports, encouraging favorable feedback while discouraging reports of substantial harms. This potential bias could have affected the recording of both benefits and adverse effects.

Lykos Therapeutics has not extensively addressed these allegations publicly. Nevertheless, a group of investigators who participated in the trials has refuted the accusations made in the ICER report, defending the integrity of their research.

Despite the setback, Lykos, previously known as MAPS Public Benefit Corporation, remains committed to collaborating with the FDA to secure approval for its MDMA-assisted therapy. The treatment's Prescription Drug User Fee Act (PDUFA) target action date is set for August 11, 2024, giving the company time to address the panel's concerns and potentially provide additional data to support their application.

In summary, while the advisory panel's decision marks a significant hurdle for Lykos Therapeutics' MDMA-assisted therapy for PTSD, the company continues to aim for FDA approval. Addressing the efficacy and ethical concerns highlighted by the advisory committee and external reviewers will be crucial for the future prospects of this treatment.