The Food and Drug Administration has rejected an application to approve MDMA for treating post-traumatic stress disorder in adults, saying more research is needed to determine its safety and efficacy.
The FDA on Friday sent a "complete response letter" to Lykos Therapeutics, the company established to develop and promote therapy using the psychedelic drug, denying approval based on concerns expressed in June by a federal advisory panel on the scientific studies of the treatment.
The panel had said the way the trials were conducted and assessed may have inflated results and raised additional questions.
The FDA agreed, saying in a statement that "significant limitations to the data contained in the application" prevented the agency from issuing conditional approval.
"The agency recognizes that there is great need for additional treatment options for mental health conditions such as PTSD," the FDA wrote. "We will continue to encourage research and drug development that will further innovation for psychedelic treatments and other therapies to address these medical needs."
The announcement is a blow to advocates, lawmakers and veterans who pushed for approval. In the weeks leading up to the FDA decision, a bipartisan group of 80 lawmakers and more than a dozen advocacy groups said the therapy was needed, given there have not been any new treatments for PTSD in more than two decades.
"Despite billions of federal dollars spent on addressing PTSD and mental health, the number of veteran lives lost to suicide has not decreased. ... This astounding lack of innovation has undoubtedly contributed to our rising veteran suicide rates over the past two decades," wrote Rep. Jack Bergman, R-Mich., and other House members in a letter to President Joe Biden on Aug. 2.
On Monday, former Navy SEAL and Republican Morgan Luttrell of Texas, said he was "deeply disappointed" in the decision and believes in the treatment's "life-changing results," given his personal experience with psychedelic therapies.
"This decision is a major setback for those who are desperately seeking and deserve effective treatment options. However, I remain committed to advancing legislation that supports innovative therapies for PTSD," Luttrell said in a statement to Military.com.
Research on the treatment has been conducted over the past decade by the Multidisciplinary Association for Psychedelic Studies, or MAPS, a nonprofit advocacy organization focused on the use of restricted medications to address medical conditions.
In a statement, Lykos, the public benefit company spun off from MAPS, said it was "deeply disappointing" for the FDA to request another Phase III clinical trial on the therapy.
"Our heart breaks for the millions of military veterans, first responders, victims of sexual and domestic abuse, and countless others suffering from PTSD who may now face more years without access to new treatment options," said Amy Emerson, the company's chief executive officer.
Emerson said the company hopes to meet with the FDA to ask for reconsideration and discuss the agency's concerns.
MAPS' studies showed that, following treatment, which involves therapy conducted with providers trained in administering the medication and coaching patients through their experiences, 87% of patients had significant improvement in PTSD systems four months after receiving treatments, and 71% no longer met the diagnostic criteria for PTSD at the end of the studies.
After the research was published, some participants said their concerns and responses were downplayed or ignored by researchers, including adverse events like increased anxiety and suicidal thoughts.
The FDA advisory panel voted 10-1 against approving the treatment based on recent trial concerns.
"It seems like there are so many problems with the data -- each one alone might be OK, but when you pile them on top of each other ... there's just a lot of questions I would have about how effective the treatment is," Melissa Decker Barone, an adjunct professor with the University of Maryland School of Medicine and advisory committee member, said during the panel discussion.
In its response to the panel, Lykos questioned the lack of subject matter experts on the panel and said the group's discussions "veered beyond the scientific content."
Lykos officials refuted arguments that prior use of MDMA, or midomafetamine, known recreationally as molly or ecstasy, colored the research and said some panelists questioned whether the psychotherapy that accompanies administration of the drug was even necessary.
"Lykos acknowledges that midomafetamine-assisted therapy represents a novel combination of drug and therapy that raises unique research questions and will continue to engage the FDA as appropriate on these challenges," they wrote.
MDMA affects patients by releasing the anxiety-reducing neurotransmitter serotonin, which allows users to think and talk through trauma without fear. It also causes increases in norepinephrine and dopamine -- two neurotransmitters associated with increase in energy, alertness and happiness, according to researchers.
Earlier this year, the VA announced funding for research into the use of psychedelic substances including MDMA and psilocybin, the active ingredient in hallucinogenic mushrooms, to treat mental health conditions.
Currently, accepted treatments for PTSD at the VA include FDA-approved medications Paxil and Zoloft, as well as prolonged exposure therapy and cognitive processing therapy.
Rep. Lou Correa, D-Calif., co-chairman of the Congressional Psychedelics Advancing Therapies Caucus, said in a statement Friday that veterans -- who die by suicide at a rate of roughly 17 each day -- could benefit from MDMA-assisted treatment.
"Today, the FDA got it wrong in a big way," Correa said in a statement. "The FDA has again chosen to ignore the science and listen to stigma and bias. This FDA decision will cost many veterans their lives."
