FDA Gives Breakthrough Therapy Status to MDMA-Assisted Psychotherapy for PTSD

Phase 3 clinical trials in patients with severe PTSD to begin in 2018

The FDA has granted breakthrough therapy designation to MDMA (midomafetamine, also known as the street-drug Ecstasy) for the treatment of posttraumatic stress disorder (PTSD). The Multidisciplinary Association for Psychedelic Studies (MAPS), a nonprofit group that develops medical, legal, and cultural contexts for people to benefit from the careful use of psychedelics and marijuana, and the FDA have also reached agreement under the special protocol assessment process for the design of two upcoming phase 3 trials.

The phase 3 trials will assess the efficacy and safety of MDMA-assisted psychotherapy in 200 to 300 participants with PTSD, 18 years of age and older, at sites in the U.S., Canada, and Israel. Participants will be randomized to receive three daylong sessions of either MDMA or placebo in conjunction with psychotherapy over a 12-week treatment period, along with 12 associated 90-minute nondrug preparatory and integration sessions. The primary endpoint will be the Clinician Administered PTSD Scale, as assessed by a blinded pool of independent raters. The first phase 3 trial will begin enrolling patients in spring 2018 after the completion of an open-label lead-in training study at phase 3 sites starting this fall.

In MAPS’ completed phase 2 trials with 107 participants, 61% no longer qualified as having PTSD after three sessions of MDMA-assisted psychotherapy two months following treatment. At the 12-month follow-up, 68% no longer had PTSD. All phase 2 participants had chronic treatment-resistant PTSD and had suffered from PTSD for an average of 17.8 years.

“Reaching agreement with FDA on the design of our phase 3 program and having the ability to work closely with the agency has been a major priority for our team,” said Amy Emerson, Executive Director of the MAPS Public Benefit Corporation. "Our phase 2 data was extremely promising with a large effect size, and we are ready to move forward quickly. With breakthrough designation, we can now move even more efficiently through the development process in collaboration with the FDA to complete phase 3.”

According to MAPS, MDMA transiently increases heart rate, blood pressure, and body temperature in a dose-dependent manner that is generally not problematic for physically healthy individuals. Serious adverse events involving administration of MDMA in MAPS studies have been uncommon and non-life threatening. Phase 2 trial results are being prepared for publication.

Source: MAPS; August 26, 2017.