US FDA declines to approve first MDMA-based PTSD treatment

NEW YORK - The United States Food and Drug Administration has declined to approve a therapy based on the mind-altering drug MDMA for patients with post-traumatic stress disorder, stating it could not be approved based on the data submitted, developer Lykos Therapeutics said on Aug 9.

Commonly known as ecstasy or molly, MDMA has long been seen by advocates as a potential treatment for mental health disorders and to have therapeutic applications beyond its illicit use.

The FDA’s decision to not approve the therapy was in line with a vote by the agency’s expert panel, which was weighing Lykos’ application. During the panel’s June meeting, advisers to the FDA had pointed to concerns around how the clinical trial was conducted.

The concerns included a “striking lack” of documentation around whether or not the trial participants had abused the drug, problems with how the trial was designed and the need for more evidence.

Lykos said the FDA has asked it to conduct an additional late-stage trial to further study the drug’s safety and efficacy.

The company plans to request a meeting with the FDA to ask for reconsideration of the decision and to discuss the agency’s recommendations for a resubmission seeking approval for the MDMA capsules.

“The FDA request for another study is deeply disappointing,” said Lykos chief executive officer Amy Emerson. “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 ... can be addressed with existing data, post-approval requirements or through reference to the scientific literature.”

California-based Lykos based its request for regulatory approval on two clinical studies, each of which enrolled around 100 people, to evaluate MDMA used together with other psychological interventions such as talk therapy, against a placebo with talk therapy.

These two studies, published in the prestigious journal Nature Medicine, indicated MDMA was indeed both safe and highly effective at treating PTSD.

But nine out of 11 experts on the FDA panel said available data was not enough to show the treatment was effective, and 10 out of 11 said the benefits did not outweigh the risks.

PTSD, a disorder caused by very stressful events, affects 13 million Americans and is commonly associated with war veterans. But natural disasters, abuse or other trauma such as rape may also trigger the condition in civilians.

Pharmaceutical treatment options for PTSD are so far limited to two antidepressants that require three months of dosing to take effect, and response rates to the medications have been found to be uneven.

MDMA – methylenedioxymethamphetamine – is a Schedule 1 drug under the Controlled Substances Act, and approving it for medical use would have represented a major shift.

While the decision is disappointing for 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 at University of California, Los Angeles’ department of psychiatry.

The regulator denying approval to Lykos’ therapy would not necessarily have a negative impact on other players in the psychedelic space because they have approached drug development and trials in slightly different ways, Evercore ISI analyst Gavin Clark-Gartner, had told Reuters ahead of the decision.

Researchers have been trying to understand how psycho-active ingredients, whether derived from cannabis, LSD or magic mushrooms, could be used to address a range of mental and behavioural health conditions, including PTSD, anxiety and depression.

The Institute for Clinical and Economic Review, an influential non-profit that evaluates clinical trials and drug prices, had said in a report in June there was insufficient evidence to show the benefits of the MDMA-assisted PTSD treatment. REUTERS, AFP