The Food and Drug Administration (FDA) on Friday denied approval of an MDMA-based treatment for post-traumatic stress disorder (PTSD), the manufacturer said, a major setback for the use of psychedelic drugs as medicine.
Drug company Lykos Therapeutics said the FDA told them the treatment “could not be approved based on data submitted to date.”
Lykos said the FDA has requested an additional Phase 3 trial to further study the safety and efficacy of the treatment, but such studies often take years. The company said it plans to request a meeting with the FDA to ask for reconsideration.
“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,” Lykos CEO Amy Emerson said in a statement.
The decision was widely expected after an independent FDA advisory panel overwhelmingly recommended against approving the treatment, which paired MDMA, commonly known as ecstasy, with talk therapy to treat PTSD.
The panel raised questions about the integrity of the trial studying the treatment. In particular, agency staff noted that patients in the trials were able to guess whether they had received MDMA or a placebo, making it virtually impossible to “blind” the study.
They praised the promise of the treatment but said the risks far outweighed any benefits, and they expressed concern that the manufacturer’s scientific work was influenced by the psychedelic evangelism of its parent organization, the Multidisciplinary Association for Psychedelic Studies. They also questioned whether the company had appropriately tracked serious adverse events during the trials.
“While conducting another Phase 3 study would take several years, we still maintain that many of the requests that had been previously discussed with the FDA and raised at the Advisory Committee meeting can be addressed with existing data, post-approval requirements or through reference to the scientific literature,” Emerson said.
The decision generated an enormous amount of lobbying from advocacy groups, including combat veterans. Earlier this week, a bipartisan group of more than 80 House and Senate lawmakers sent letters to FDA and the White House urging the approval.
The FDA has not approved any new treatments for PTSD in more than two decades, and proponents have said continued high rates of veteran suicide mean the government must do more to protect veterans.
A growing body of research has shown that psychedelics like LSD, psilocybin mushrooms and MDMA can have significant positive impacts on conditions like PTSD.
The application marked the first time the U.S. has considered a Schedule I drug — those considered to have a high potential for abuse such as heroin and LSD — for medical use.
More than 13 million individuals in the U.S. live with PTSD, and veterans are disproportionately affected, according to estimates from the National Center for PTSD. But current treatments are notoriously not very effective.