Doria Keys
Associate Editor
Loyola University Chicago School of Law, JD 2025
Anxiety disorders are among the most common mental illnesses diagnosed in the United States. Specifically, General Anxiety Disorder (GAD) manifests as persistent and exaggerated worry about a number of different things in life, including, but not limited to, finances, health, family, and employment. GAD affects about 6.8 million adults across the nation in any given year; however, according to the Anxiety & Depression Association of America, only 43.2% of those affected are receiving treatment for GAD. Despite how common GAD is, there has been little innovation in treatment for the past two decades. In fact, the last new drug approval for the treatment of GAD was approved in 2004.
Recently, the Food and Drug Administration (FDA) granted Breakthrough Therapy Designation (BTD) status to MM120, a form of the psychedelic drug lysergide d-tartrate (LSD), for treatment of GAD. MM120 is currently being developed by MindMed, a biopharma company dedicated to “developing psychedelic inspired medicines that seek to treat underlying causes of distress in the brain.” MM120 has seen promising results for treating GAD, showing rapid and sustained clinical improvement for 65% of patients within three months.
What is Breakthrough Therapy Designation (BTD)?
Breakthrough Therapy Designation is an expedited program by the FDA available to a drug that is intended to treat a serious or life-threatening disease or condition when initial clinical data suggests that the drug may present considerable advancements compared to current treatments on one or multiple clinically relevant measures. The premarket development duration for breakthrough therapies is typically two to three years less than that for non-designated therapies, and the review process for applications is shortened by several months.
The FDA’s BTD is not the same as a full drug approval. It does not grant marketing authorization or make the drug immediately available for use by the general public. Instead, what it signifies is the FDA’s recognition that the drug, in this case, MM120, has the potential to address an unmet medical need. The FDA’s BTD of MM120 is based on the substantial need for effective treatments for GAD. Currently, there is a large unmet need for individuals living with GAD and not responding to FDA-approved treatments.
Regulation of psychedelics in the United States
The Controlled Substances Act, which can be found at 21 U.S.C. 13, has labeled psychedelics as Schedule I substances, meaning that they have a high potential for abuse and no currently accepted medical use in the United States. Therefore, it is currently prohibited to manufacture, import, use, or distribute psychedelic substances.
The only type exception to the Controlled Substances Act can be found in the scope of the Religious Freedom Restoration Act of 1993, which allows psychedelics to be used for religious ceremonies in certain circumstances. However, this protection is not guaranteed.
In response to nationwide efforts to remove regulatory barriers on the use of psychedelics, on June 23, 2023, the FDA released a Draft Guidance to offer overarching considerations for sponsors, such as MindMed, engaged in the development of psychedelic medications to treat various medical conditions, such as psychiatric disorders and substance use disorder. As with any drug, the manufacturing of psychedelics must comply with current good manufacturing practice (CGMP) included in section 501(a)(2)(b) of the Federal Food, Drug, and Cosmetic Act. In addition, because psychedelics are Schedule I controlled substances, the United States Drug Enforcement Administration (DEA) closely monitors their distribution and use. Any research involving psychedelic substances must comply with DEA regulations to ensure safety and prevent unauthorized access or distribution. Researchers conducting studies with these substances must obtain specific licenses and register with the DEA, and their facilities must meet strict security and record-keeping requirements. Compliance with these regulations is essential for obtaining approval to conduct clinical trials and research involving Schedule I substances, including psychedelics.
What does this mean for the future of medical treatment with psychedelics?
In addition to LSD, FDA has granted Breakthrough Therapy Designation to two other psychedelics: (1) MDMA (3,4 methylene-dioxymethamphetamine), commonly known as ecstasy or molly, for treatment of post-traumatic stress disorder (PTSD), and (2) psilocybin, commonly known as ‘magic mushrooms’, for treatment-resistant depression and major depressive disorder (MDD).
Currently, under federal law, LSD, MDMA, and psilocybin are classified as Schedule I drugs under the Controlled Substances Act, meaning that they have a high potential for abuse and no currently accepted medical use. However, in light of recent studies acknowledging their safety and effectiveness in treating anxiety and depression, the industry is pushing for reclassification of psychedelics due to their therapeutic potential. As of July 1, 2023, Australia has become the first country in the world to reschedule MDMA and Psylocibin from ‘prohibited’ to ‘controlled’ for treating specific mental health conditions. In addition, state legislative reforms are shifting prospects of psychedelic treatment and nationwide legalization. The future of psychedelics in the United States will largely be determinative of the outcomes and FDA decisions on the ongoing clinical trials and drug approvals. FDA’s expedited approval process for several forms of psychedelics show a hopeful future of reclassification and implementation of psychedelics for medical treatment.