The US Food and Drug Administration has rejected a first-of-its-kind proposal to use the psychedelic drug MDMA, also known as ecstasy or Molly, as a treatment for post-traumatic stress disorder.
Drugmaker Lykos Therapeutics had asked the FDA to approve the drug in combination with talk therapy. The company said Friday that the regulatory agency has requested an additional Phase 3 trial so that the safety and efficacy of the therapy can be further studied. The decision comes after an FDA advisory panel in June concluded that there wasn’t enough evidence to recommend its approval.
Lykos said it plans to request a meeting with the FDA to ask for reconsideration of the decision and to further discuss the agency’s recommendations. “The FDA request for another study is deeply disappointing, not just for all those who dedicated their lives to this pioneering effort, but principally for the millions of Americans with PTSD, along with their loved ones, who have not seen any new treatment options in over two decades,” said Lykos CEO Amy Emerson in a company statement. She added that conducting another Phase 3 trial would take several years.
As many as 13 million Americans suffer from PTSD in any given year, according to the National Center for PTSD. Just two drugs have been specifically approved to treat the disorder, with the latest being greenlit by the FDA in 2000. The lack of options has turned combat veterans into unlikely advocates for MDMA-assisted therapy. In the days leading up to the FDA decision, veterans groups and members of Congress from both parties pressed for its approval.
In a letter to President Biden, more than 300 veterans and representatives of veterans service organizations wrote that MDMA-assisted therapy “offers desperately needed hope for veterans and their families, with the potential to save and drastically improve countless lives over the coming years.”
A bipartisan group of more than 60 members of the House of Representatives and 19 senators also voiced their support for the therapy this week. “Given the substantial burden of PTSD and the current treatment limitations, the possibility of new, more effective therapies is particularly meaningful,” the lawmakers wrote in a letter to FDA commissioner Robert Califf.
There has been increasing interest in recent years in using MDMA and other psychedelics to treat severe mental illness. But with the FDA decision, MDMA will remain a federally prohibited substance listed as Schedule I drug, defined as those that have “no currently accepted medical use and a high potential for abuse.”
During a nine-hour meeting on June 4, Lykos representatives made their case to an independent panel of FDA advisers. The company’s clinical trial data showed that more than 86 percent of study participants who underwent MDMA-assisted therapy experienced a measurable reduction in the severity of their PTSD symptoms, and 71 percent improved enough that they no longer met the criteria for a diagnosis. In a placebo group, 69 percent improved and nearly 48 percent no longer qualified for a PTSD diagnosis.
Despite the positive results, advisory committee members cited concerns about the reliability of the clinical trial data, the long-term efficacy of the treatment, and the standardization of the talk therapy given during the MDMA sessions. One major question raised by the advisory committee was the extent to which the talk therapy contributes to the treatment benefit.
The panel overwhelmingly voted that there wasn’t enough evidence to recommend approval. Just two of the 11 committee members agreed that MDMA-assisted therapy is effective based on the evidence Lykos presented, and only one thought its benefits outweighed the risks. The FDA usually follows the recommendations of advisory committees but is not obligated to do so.
Lykos said it will “work diligently in the coming months to address FDA’s concerns and to take advantage of agency processes to resolve scientific disagreements.”