FDA Rejects Lykos’ Drug Application and Requests an Additional Phase 3 Clinical Trial

Late last week, Lykos Therapeutics announced that it had received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding its application for registration of midomafetamine (MDMA) as a new drug application (NDA) for the treatment of post-traumatic stress disorder (PTSD)1.

In its letter, the FDA noted that some critical data regarding the safety and efficacy of MDMA are currently missing, so the FDA cannot approve the NDA in its current form. Instead, the FDA has suggested that Lykos conduct an additional Phase 3 clinical trial, focusing on collecting the data that the agency believes are presently missing.

Overall, the comments on the NDA are based on the findings of an independent advisory committee to the FDA, which we previously reported on 2. Lykos indicated that it will request the FDA to reconsider the decision according to the agency's established procedures. Firstly, Lykos believes that the existing experimental data should be sufficient to assess the safety and efficacy of MDMA in the treatment of PTSD. Secondly, Lykos emphasizes that additional research will take several years at least. During this time, patients will not have access to treatment. Moreover, Lykos still has to find the funds to conduct this study. This can be quite difficult, as MDMA is not eligible for patent protection. As a result, profit-oriented companies are not particularly motivated to invest in such research.

The current position of the FDA is not a final denial of approval, but indicates the need for further research before the drug can be approved for widespread market introduction. In any case, Lykos promises to inform the public about its further plans after the meeting with the FDA.

For the global community, this news means either waiting for the new trial to be conducted in the U.S. or investing in their own scientific initiatives. It is entirely possible that Europeans, rather than Americans, will lead the way in rolling out MDMA-assisted therapy. For example, an expert commission recently recommended that the Dutch government introduce MDMA-assisted therapy for PTSD3. Shortly before that, the EU government allocated a record 6.5 million euros for psilocybin-assisted psychotherapy research under the Horizon Europe program 4. And more recently, the European Medicines Agency (EMA) published a report on the prospects for implementing psychedelic-assisted therapy in the EU, emphasizing the need for collaboration between scientists and governmental institutions to develop this promising and innovative field 5.

The FDA's decision once again confirms that Ukraine cannot afford to rely solely on foreign researchers. Even if the FDA is eventually satisfied with the results of the new Lykos study, it will take several years to complete. Even more time will pass between U.S. approval and accessibility in Ukraine. There is no need to reiterate why Ukraine urgently needs effective treatments for PTSD and other stress-related disorders now, not in 10 years. This is why we emphasize the need to develop our own research and development capacity to ensure timely access to innovative treatments for patients. First and foremost, the Ministry of Health should approve the regulatory framework already mandated by law that would regulate the research on the therapeutic use of psychedelics. The rest we can handle ourselves.