The FDA’s upcoming Pharmacy Compounding Advisory Committee meeting on unapproved peptides is about more than a handful of substances under review. At stake is whether our regulatory system will still enforce the line between unproven, experimental treatments and proven medicines – or let one be mistaken for the other.
Researchers are investigating various peptides for a range of conditions, from wound healing to metabolic and neurological disorders. That work deserves support. But innovation and potential promise are not the same as scientific proof, and research into possible uses should not be mistaken for clinical readiness.
Several of the peptides before the Committee illustrate exactly why rigorous evaluation matters. Emideltide (also known as DSIP), proposed to treat conditions including chronic insomnia and narcolepsy, has already been studied in human trials with only a handful of participants. Those studies found no meaningful improvement over placebos in objective measures of sleep or patients’ reported sleep quality. FDA reviewers concluded that the available evidence was too limited—and the studies too small and uncontrolled—to establish a meaningful clinical benefit.
For several other peptides under consideration, the evidence is even more limited. MOTS-c has been promoted for obesity, healthy aging, and metabolic health, yet FDA reviewers identified no published human clinical studies supporting these therapeutic uses – and the same was true for KPV. BPC-157 has gained widespread attention online for purported tissue healing despite a lack of adequate clinical evidence demonstrating that it is safe and effective for patients, while TB-500 raised concerns about questionable formulations and potential immune reactions without sufficient human safety data.
Although Semax and Epitalon have been studied internationally, the FDA concluded that the available evidence does not establish their safety or efficacy. In the case of Epitalon, FDA scientists also highlighted unresolved concerns about cancer risk, underscoring the need for more robust safety data before it reaches patients.
Unfortunately, marketing of these peptides has outpaced the science – and it is patients who will pay the price. Online advertising, social media testimonials, and wellness clinics increasingly market these peptides as established therapies, manufacturing the illusion that clinical and regulatory review is an unnecessary bureaucratic formality rather than an important safeguard. That’s not how a system designed for patient safety should work: Scientific evidence should drive clinical use—not hype and not the marketplace.
This is also why pharmacy compounding must remain true to its intended purpose. Congress created narrow compounding exemptions to meet the unique medical needs of individual patients when FDA-approved medicines cannot. It did not create an alternative pathway for broad access to experimental therapies with uncertain safety and efficacy. Allowing compounding to create that pathway risks blurring the line between individualized patient care and drug development, ultimately asking patients to accept undue risks without adequate safety screening.
The FDA’s responsibility is not to determine whether these peptides are exciting or popular; it is to determine whether the available evidence supports their use within the narrow compounding framework established by Congress. Maintaining that standard protects patients today without compromising public confidence in the scientific process that can deliver the future’s innovative therapies.
Ronald P. Jordan, RPh is Former President of the American Pharmacists Association and Dean Emeritus of the Chapman University School of Pharmacy
Philip Schneider, MS, FASHP, FASPEN, FFIP is Professor of Pharmacy at The Ohio State University and Advisory Board Chairman of Alliance for Safe Biologic Medicines
