The Food and Drug Administration (FDA) has chosen not to approve Lykos Therapeutics' MDMA-assisted therapy for post-traumatic stress disorder (PTSD), the company said on Friday.
The FDA said Lykos’ new drug application for midomafetamine capsules for PTSD could not be approved at this time based on the data submitted to date and has asked the company to complete an additional Phase 3 study, Lykos said in a statement.
Lykos plans to request a meeting with the regulator to ask for reconsideration of the decision.
Issues raised by the FDA in its decision mirror those raised during an FDA Advisory Committee Meeting on June 4, which included concerns that Lykos' clinical data were insufficient to demonstrate durability along with questions about expectancy bias stemming primarily from participants with prior MDMA use.
Lykos has previously published its response to the issues discussed at the meeting and reiterated that it believes the data included in its new drug application provided “sufficient evidence of efficacy and durability” in line with the relevant FDA guidance.
"The FDA request for another study is deeply disappointing, not just for all those who dedicated their lives to this pioneering effort, but principally for the millions of Americans with PTSD, along with their loved ones, who have not seen any new treatment options in over two decades," Lykos CEO Amy Emerson said.
"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 and raised at the Advisory Committee meeting can be addressed with existing data, post-approval requirements or through reference to the scientific literature."
Dr Jennifer Mitchell, Professor of Neurology and Psychiatry and Behavioral Sciences at the University of California San Fransisco, said the FDA’s decision to request another Phase 3 study was "a major setback" for the field.
"Developing the first-ever clinical trial design to assess the safety and efficacy of midomafetamine-assisted therapy with patients suffering from moderate to severe PTSD is an enormously complex undertaking," Dr Mitchell said.
"Over the course of many years, the researchers, with the support of our sponsor, Lykos, developed and executed Phase 3 studies that we believe demonstrated the approvability of this treatment. We did so in consultation with the FDA and with an agreed Special Protocol Assessment in place.”