The US Food and Drug Administration (FDA) has granted breakthrough therapy designation to MDMA (3,4-methylenedioxymethamphetamine) as an adjunct to psychotherapy for adults with posttraumatic stress disorder (PTSD).
According to the Multidisciplinary Association for Psychedelic Studies (MAPS), the FDA has agreed on the design of two upcoming phase 3 trials of the drug, also known as ecstasy, in patients with severe PTSD.
This agreement was reached under the FDA's Special Protocol Assessment Process and "confirms that the protocol design, clinical endpoints, planned conduct, and statistical analyses for the phase 3 trials...are acceptable to support regulatory approval," MAPS notes in a press release.
The organization reports that the first of these trials, known as MAPP1, is set to start enrollment in spring 2018. However, an open-label lead-in training study will begin this fall.
"For the first time ever, psychedelic-assisted psychotherapy will be evaluated in phase 3 trials for possible prescription use," Rick Doblin, PhD, founder and executive director of MAPS, said in the release.
"Now that we have agreement with FDA, we are ready to start negotiations with the European Medicines Agency," he added.
Promising Phase 2 Results
Breakthrough therapy designation from the FDA means that a treatment has a "meaningful advantage" compared with existing medications in treating serious or life-threatening conditions. Also, previous yet preliminary research must show substantial improvement for the novel treatment over those that are currently available.
Completed phase 2 trials from MAPS included 107 patients with treatment-resistant PTSD (mean duration, 17.8 years). Combined results showed that after three sessions of MDMA-assisted psychotherapy, 61% of the participants no longer met PTSD criteria 2 months post treatment. This number increased to 68% 1 year post treatment. Full data from these trials are expected to be published soon.
The upcoming, randomized, phase 3 MAPP1 trial will evaluate 100 to 150 patients with PTSD at centers in the United States, Canada, and Israel. The primary outcome measure will be improvement in scores on the Clinician Administered PTSD Scale, which is known as the CAPS-5.
If the results of this and the phase 3 MAPP2 trial show significant efficacy and safety, the organization said MDMA-assisted psychotherapy "is on track for FDA approval by 2021."
Although MDMA, in a dose-dependent manner, has been found to transiently increase blood pressure, heart rate, and body temperature, this was "generally not problematic for physically healthy individuals," according to MAPS.
Medscape Medical News © 2017
Cite this: FDA Okays Clinical Trial Testing of Psychedelic Drug for PTSD - Medscape - Aug 30, 2017.