The psychedelic drug MDMA failed to get backing from U.S. regulatory advisers to treat a form of mental illness, a setback to patients and advocates pushing for better treatment options.
Advisers to the Food and Drug Administration voted 9 to 2 that developer Lykos Therapeutics Inc.’s data failed to show the drug is effective in patients with post-traumatic stress disorder. The panel also voted 10-1 that a safety program wouldn’t be sufficient to protect patients from potential risks of the therapy.
The FDA will spend the next several weeks deciding whether to grant clearance for the medication before the deadline of August 11. The agency doesn’t have to follow the counsel of its advisers, but it frequently does. In cases when the FDA decides not to approve, the agency and the sponsor discuss potential next steps such as providing additional data.
The strongly negative vote puts the regulator in a tough spot. Some of the panel’s efficacy concerns relate the design of Lykos’ clinical trials, which the agency signed off on back in 2017. The committee had concerns across the board, from how the trials was designed and conducted to the potential for vulnerable patients to be harmed or abused. Worries also arose about MDMA-related cardiac side effects and the potential an approval would lead to increased diversion of MDMA for recreational use.
“There’s a lot of holes in this data set,” Rajesh Narendran, a psychiatrist from the University of Pittsburgh Medical Center who chairs the panel, said during a discussion period. “I’m not convinced at all this drug is effective based on the data I saw.”
The FDA has been under pressure to approve medications that treat PTSD, a mental illness that sometimes develops after patients witness or experience a life-threatening or traumatic event. Some 13 million Americans have PTSD in any given year, according to the Department of Veterans Affairs, and the few treatment options include two approved drugs that aren’t effective for many people.
With its large PTSD patient population, the VA has been “proactively preparing for a potential approval of MDMA-assisted therapy by the FDA,” a spokesperson said in an email last month. The government agency has already put together a task force to prepare for the MDMA rollout in veterans.
Companies including Mind Medicine Inc., Atai Life Sciences and Compass Pathways are developing psychedelic drugs for medical purposes. Clearance of MDMA, known more commonly as ecstasy or molly, could add to the push for recreational drugs into mainstream applications. Shares of Mind Medicine fell 2.5% after U.S. markets closed, while Compass Pathways’ fell 9% in after-market U.S. trading following the negative vote.
Powerful drugs
Psychedelics are powerful drugs that affect mood, perception and cognitive processes. Currently MDMA is deemed a schedule 1 drug by the Drug Enforcement Administration, on the same level as heroin and LSD, meaning it has high risk for abuse or addiction. Schedule 3 drugs like ketamine and anabolic steroids, which have medical uses, have a lower risk.
Critics have raised concerns about whether studies of MDMA can be taken at face value. A key issue is that patients may be aware of whether they received the drug or a placebo because of MDMA’s pyschedelic effects. There are also controversies around underreported patient safety events and questions about whether therapists involved in the trials were too invested in its success.
“We already have evidence-based treatments for PTSD,” said Melissa Barone, a psychologist with the VA Maryland Health Care System who sits on the panel. “There’s just a lot of questions still, that I would have about how effective the treatment is and how durable it is.”
In documents published ahead of the meeting, FDA staff said that “several factors make these data challenging to interpret and complicate the benefit-risk assessment.”
The drug may also raise the risk of heart attacks and strokes by increasing patients’ pulse and blood pressure, the FDA staff said in their report, and cited the vulnerability of patients under the influence of the drug.
“We are particularly concerned that serious harm could result while patients are impaired,” they wrote.
Other medical groups, including the Institute for Clinical and Economic Review and review panel of the American Psychological Association, have said that there’s insufficient data to show the benefit of MDMA for PTSD treatment. The ICER report also said study participants may have felt pressured to report good outcomes and suppress bad ones, and acknowledged an allegation of sexual misconduct during an early trial.
“We certainly take those allegations very seriously and are quite concerned by them,” Tiffany Farchione, FDA’s psychiatry division director, said in the meeting. “We do have inspections ongoing at this point.”
Lykos’ drug is taken in studies in conjunction with 42 hours of talk therapy, including three daylong sessions during which patients are under the influence of the drug.
If the FDA acts against the panel’s advice and approves the drug, it could take up to three months for the U.S. Drug Enforcement Administration to move it from the strictest category of controlled substances to a less restrictive one. And even then, the process is further complicated by state laws.
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