Updated August 09, 2024 at 19:11 PM ET
On Friday, drugmaker Lykos Therapeutics from the Food and Drug Administration that the agency has decided not to approve MDMA-assisted therapy for PTSD at this time. Instead, the agency asked the drugmaker to further study the safety and efficacy of the treatment.
The heavily anticipated announcement came in the form of a 鈥渃omplete response letter鈥� from the FDA to Lykos, according to a company press release.
It represents a significant setback for the company and the broader movement to bring psychedelics into the mainstream of mental health care.
鈥淚t鈥檚 a huge blow to the field,鈥� says , an anesthesiologist at Stanford University whose lab studies psychedelics.
Lykos said it plans to request a meeting with the FDA to ask for reconsideration of the decision.
Lykos鈥� CEO Amy Emerson called the FDA鈥檚 request for another phase 3 trial 鈥渄eeply disappointing,鈥� and said conducting the study 鈥渨ould take several years.鈥�
She added that many of the requests from the agency 鈥渃an be addressed with existing data, post-approval requirements or through reference to the scientific literature."
The treatment had garnered considerable support from patients, leaders in the field of mental health and psychedelics and politicians from both parties. In recent weeks, advocates campaign, emphasizing the dire need for new and effective treatments for veterans and the millions of people affected by PTSD.
Many in the psychedelics industry had viewed this as a pivotal moment. The FDA decision on MDMA could be seen as a bellwether for other drugs that are in the pipeline like psilocybin and LSD, which are both considered 鈥渃lassic psychedelics鈥� unlike MDMA.
But Dr. Mason Marks believes the FDA鈥檚 decision doesn鈥檛 spell trouble for the broader field because the agency has signaled repeatedly it鈥檚 open to psychedelic research.
鈥淚 think this is a unique case,鈥� says Marks, a law professor and senior fellow with the Project on Psychedelics Law and Regulation at Harvard Law School's Petrie-Flom Center.
鈥淚 don't think there's any sign that progress is going to be slowed. If anything, it might intensify because the other companies might see an opportunity to really get in there and compete,鈥� he says.
The wave of enthusiasm around MDMA's therapeutic potential has come from research scientists at top academic centers and even the Department of Veterans Affairs.
Supporters of the drug were dismayed by Friday's decision.
鈥淭he FDA鈥檚 decision is disgraceful,鈥� said Heroic Hearts Project, a veterans organization that had lobbied for FDA approval citing the many veteran suicides in a statement. 鈥淭his is the epitome of bureaucratic red tape 鈥� and the result is people will keep dying.鈥�
Opposition to the Lykos鈥� application had gained momentum in recent months, culminating in a contentious public meeting when advisors to the FDA backing the treatment, largely based on concerns about shortcomings in the clinical research.
In its press release, Lykos said the FDA echoed the issues raised by the committee.
The committee's downvote put the FDA in a tricky spot, given that it historically sides with the advisors.
鈥淎s discussed at the advisory committee meeting, there are significant limitations to the data contained in the application that prevent the agency from concluding that this drug is safe and effective for the proposed indication,鈥� an FDA spokesperson said to NPR in an email.
鈥淲e will continue to encourage research and drug development that will further innovation for psychedelic treatments and other therapies.鈥�
Those hoping to see MDMA make it to market argue that concerns about the quality of the research were overblown and, in some cases, focused on issues with the study design that weren鈥檛 unique to MDMA.
The trials 鈥渦ndoubtedly had problems,鈥� says Stanford's , but the FDA could have dealt with those concerns by approving the treatment with strict restrictions around how it鈥檚 administered and a requirement to do a post-market study.
He worries the decision to deny approval will stifle innovation and funding into other novel treatments, including MDMA-like drugs that are being developed.
鈥淭he FDA has gone against advisory committee advice [in the past]. It has approved drugs with abuse potential,鈥� says Heifets. 鈥淣one of this is that radical, so I think they could have done something else here.鈥�
On the other hand, Marks says that approval would have required the FDA to 鈥減ile on鈥� so many restrictions because of concerns about the Lykos鈥� application that ultimately it would have been 鈥渋mpractical鈥� for the treatment to reach many people.
He also wonders whether the all-out publicity blitz by Lykos and its allies was, in the end, 鈥渃ounterproductive鈥� because it put the FDA in an awkward position by making the decision so politicized.
鈥淭his is not the end of the road for Lykos,鈥� he says, 鈥淚t will cost them financially, but it doesn鈥檛 mean they can鈥檛 ultimately succeed.鈥�
The push to establish MDMA-assisted therapy as a legitimate treatment for PTSD stretches back two decades.
Researchers affiliated with the nonprofit Multidisciplinary Association for Psychedelic Studies, or MAPS, pioneered the early studies and developed the therapy protocol embedded in the treatment. That work ultimately set the stage for the drugmaker, which was incubated by MAPS, to press forward with larger human trials and seek approval from federal regulators.
Lykos has more than $100 million in anticipation of FDA approval of its MDMA therapy.
Optimism around the treatment largely stems from the company鈥檚 promising phase 3 clinical trials, which together enrolled about 200 people. The most recent one, published last year, showed no longer met the diagnostics criteria for PTSD after three therapy sessions with MDMA, compared to about 48% who had the same talk therapy protocol but took a placebo.
Follow-up research from the company showed participants were still benefiting from the treatment at least half a year after their last dosing session.
鈥淎lthough disappointing, this move by the FDA highlights the importance of conducting rigorous research into the safety and efficacy of MDMA-assisted therapy,鈥� says , director of the Center for Psychedelic Drug Research and Education at the Ohio State University, 鈥淒espite this setback, I am hopeful that there will be a better outcome down the road after more research is completed in the coming years.鈥�
Debate over the merits of the Lykos鈥� application has become increasingly divisive, that misconduct and bias in the clinical trials compromised the findings and undermined its safety. The drugmaker and many of the therapists involved in the research have steadfastly denied those claims.
It鈥檚 unclear to what extent any of this factored into the FDA鈥檚 deliberation on the therapy.
In response to Friday鈥檚 decision, Emerson reiterated the company鈥檚 plan to push the research forward, saying they鈥檇 鈥渨ork tirelessly and use all available regulatory pathways to find a reasonable and expeditious path forward for patients.鈥�
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