This decision follows a mixed response from the FDA advisory committee, which voted against recommending the treatment. However, the need for new PTSD treatments remains urgent, and some experts are optimistic about its potential impact.
Currently, more than 13 million people in the US suffer from PTSD, including many veterans and victims of domestic and sexual abuse. Existing treatments are often ineffective, with 40% to 60% of patients not achieving remission. The FDA has not approved a new PTSD treatment in nearly 25 years, highlighting the significant unmet medical need.
Lykos CEO Amy Emerson emphasized the company’s commitment to providing new treatment options. “We have conducted significant scientific research, including two phase 3 trials required by the FDA for new treatments,” Emerson said.
These trials, MAPP1 and MAPP2, demonstrated significant reductions in PTSD severity among patients treated with MDMA-assisted therapy compared to those receiving a placebo. Common side effects included muscle tightness, nausea, decreased appetite, and sweating, which were mostly mild or moderate.
Despite these promising results, the FDA’s Psychopharmacologic Drugs Advisory Committee (PDAC) raised concerns about the methodology of the trials, including issues of selection bias and functional unblinding. Functional unblinding, a known challenge in psychiatric drug trials, was addressed extensively with the FDA during the Special Protocol Assessment (SPA) process. “We took many steps to minimize its impact, including the use of independent, blinded third-party clinician raters to assess outcomes,” Emerson added.
Another point of contention was the prior use of illicit MDMA by some study participants. However, Lykos' data indicated no meaningful differences in outcomes between participants with or without prior MDMA use. “The data shows that prior illicit MDMA use had no impact on the results,” Emerson said.
Concerns about the regulation of psychological interventions if the treatment is approved were also discussed. Emerson explained that psychotherapy is already a regulated field with oversight provided by licensing boards and professional societies. “We expect healthcare professionals to utilize their clinical judgment to deliver treatment effectively, with appropriate oversight.”
The PDAC also raised questions about potential cardiovascular and liver toxicity risks. Lykos plans to continue working with the FDA to address these concerns, possibly by collecting additional data. “We are confident that these risks can be safely addressed in a post-marketing environment,” Emerson said.
Despite the advisory committee's vote, some experts believe the FDA may still approve the therapy due to the substantial need for new PTSD treatments and the possibility of addressing the committee’s concerns. Jerry Rosenbaum, a professor of psychiatry at Harvard Medical School, highlighted the high dropout rates with existing therapies and the demonstrated efficacy of MDMA-assisted psychotherapy for PTSD.
As Lykos moves forward, Emerson reaffirmed the company’s dedication to thorough and transparent processes. “We have full confidence in the rigor and thoughtfulness that the FDA will give our application,” she said. The company is also prepared to explore the potential of MDMA-assisted therapy for other psychiatric disorders if approved.
The upcoming FDA decision is eagerly anticipated, with the potential to offer a new lifeline for millions of PTSD sufferers. Lykos remains committed to addressing any remaining questions and providing the necessary data to support the approval of midomafetamine.
