Lykos Therapeutics announced that the FDA has issued a complete response letter (CRL) for its new drug application (NDA) for midomafetamine capsules, a treatment for post-traumatic stress disorder (PTSD) in adults. The FDA stated that the application could not be approved based on the data submitted and requested an additional Phase 3 trial to further study the safety and efficacy of the drug.
The issues raised in the CRL align with concerns discussed during the FDA Advisory Committee meeting on June 4, 2024. These concerns included the durability of the treatment effect and potential expectancy bias stemming from participants' prior MDMA use. Lykos believes that the data included in the NDA provide sufficient evidence of efficacy and durability, consistent with FDA guidance. The company had aligned with the FDA in a Special Protocol Assessment (SPA) in 2017 on bias minimization measures.
Disappointment and Next Steps
Amy Emerson, CEO of Lykos Therapeutics, expressed deep disappointment, stating, "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...who have not seen any new treatment options in over two decades." Lykos plans to engage with the FDA to address their concerns and explore available regulatory pathways.
Background on Midomafetamine-Assisted Therapy
Midomafetamine is a drug commonly known to mental health professionals. Lykos has pioneered clinical trials evaluating the efficacy and safety of MDMA-assisted therapy, combining midomafetamine with psychological intervention. The company submitted an NDA to the FDA seeking approval for the treatment of PTSD in adults, which was granted Priority Review on February 9, 2024.
The Burden of PTSD
PTSD affects approximately 13 million Americans each year, with women and marginalized groups being disproportionately affected. The condition results in debilitating symptoms, impacting interpersonal relationships and overall quality of life. PTSD is often comorbid with anxiety, depression, and substance use disorders, leading to an estimated annual cost of over $232 billion in the United States.
Current Treatment Landscape
First-line treatments for PTSD include trauma-focused talk therapy, often combined with medications like sertraline and paroxetine (SSRIs). However, many individuals do not respond adequately to these treatments, underscoring the need for new evidence-based therapies. There have been no new drug treatments approved by the FDA for PTSD in over twenty years.
Lykos' Commitment
Despite the setback, Lykos remains committed to developing midomafetamine-assisted therapy for PTSD. The company will work to address the FDA's concerns and find a path forward for patients who could benefit from this treatment approach.
