US FDA rejects approval of first MDMA-based PTSD treatment

The US Food and Drug Administration (FDA) has rejected approval of a therapy based on the mind-altering drug MDMA for patients with post-traumatic stress disorder (PTSD) and stated that approval was not possible based on the data submitted, developer Lykos Therapeutics announced on Friday.

MDMA, commonly known as ecstasy or molly, has long been viewed by advocates as a potential treatment for mental disorders and also has therapeutic applications beyond its illicit use.

The Food and Drug Administration's decision not to approve the therapy was consistent with the vote of the agency's expert panel that reviewed Lykos' application. During the panel's meeting in June, FDA advisers raised concerns about the conduct of the clinical trial.

Concerns included a “glaring lack” of documentation on whether or not study participants had abused the drug, problems with the study's design and the need for further evidence.

Lykos said the FDA has asked the company to conduct an additional late-stage study to further evaluate the drug's safety and effectiveness.

The company plans to request a meeting with the FDA to request a review of the decision and to discuss the agency's recommendations for resubmitting a new application for the MDMA capsules.

“The FDA's request for another study is deeply disappointing,” said Amy Emerson, CEO of Lykos. “Although conducting another Phase 3 study would take several years, we believe that many of the requests previously discussed with the FDA can be met with existing data, post-approval requirements or by reference to the scientific literature.”

The drug, consisting of MDMA capsules from the non-profit company Lykos, should be administered together with talk therapy sessions by a licensed psychotherapist.

Lykos enrolled more than 190 patients in two late-stage trials who received either MDMA doses or a placebo in addition to therapy. The company said most study participants experienced a significant reduction in PTSD scores compared to placebo.

PTSD, a disorder triggered by highly stressful events, affects 13 million Americans and is often associated with war veterans. But natural disasters, abuse, or other trauma can also trigger the condition in civilians.

While the decision may be disappointing to some, “the additional data and requirements requested by the FDA are ultimately intended to improve their understanding of the treatment,” said Walter Dunn, assistant professor in the department of psychiatry at the University of California, Los Angeles.

The fact that the regulator did not approve Lykos' therapy would not necessarily have a negative impact on other players in the psychedelic space, as they take slightly different approaches to developing and testing drugs, Evercore ISI analyst Gavin Clark-Gartner told Reuters before the decision.

Researchers are trying to understand how psychoactive ingredients – whether derived from cannabis, LSD or magic mushrooms – could be used to treat a range of mental and psychological conditions, including PTSD, anxiety and depression.

The Institute for Clinical and Economic Review, an influential nonprofit organization that evaluates clinical trials and drug prices, said in a report in June that there was insufficient evidence to support the benefits of MDMA-assisted PTSD treatment.