US FDA rejects use of psychedelic MDMA for PTSD patients

US regulators rejected the use of Lykos Therapeutics Inc.’s psychedelic drug MDMA for post-traumatic stress disorder and asked for more data, a setback to the nascent field that’s won supporters across the political and corporate spectrum.

The Food and Drug Administration, which has been under pressure to approve treatments for PTSD, told the drugmaker that another large-scale study is needed before it could be sold. The mental health condition that can develop after life-threatening or traumatic events affects roughly 13 million Americans and has few effective treatment options.

Widespread suffering from the condition, particularly among the nation’s veterans, coupled with promising early results created a groundswell of support for the therapy. But questions quickly arose about whether the groundbreaking treatment would be used safely and appropriately, and whether the initial results were reliable.

“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,” the FDA said in an emailed statement.

Request for Reconsideration

The closely held company is pushing back on the agency’s request, saying it would take an inordinate amount of time to complete the work. It plans to request a meeting with the agency to ask for reconsideration of the decision.

The request for another study is “deeply disappointing,” Lykos Chief Executive Officer Amy Emerson said in a statement. It would take several years to complete the type of definitive trial the agency requested, she said.

The issues the FDA raised in the complete response letter it sent to Lykos echoed those raised during the advisory committee meeting in June. The agency was widely expected to delay or outright reject the application following that meeting, when the advisers to the FDA voted against using MDMA.

They concluded that Lykos hadn’t proved that the drug was effective, saying patients could tell if they were getting MDMA therapy instead of a placebo and may have fared better as a result. The advisers also cited limited information regarding adverse events and potential clinical trial misconduct.

The Multidisciplinary Association for Psychedelic Studies — the non-profit organization behind Lykos — said in a statement that it was “evaluating the path forward for safe, legal access to MDMA-assisted therapy.”

Shot in the Gut

Advocates had hoped the treatment, more commonly known as ecstasy or molly, would become the first new drug for PTSD in more than two decades.

“This is a shot in the gut, it just takes the wind out of you as a veteran,” said Jesse Gould, a 37-year-old former Army Ranger diagnosed with PTSD, who has advocated for MDMA therapy through his Heroic Hearts Project. “The FDA has the luxury of time to delay this, but I don’t, and nor do the vast majority of veterans in this country as the suicide epidemic continues unaddressed.”