The FDA is looking to get thrifty with its approved drugs. Through a newly proposed repurposing initiative, the agency hopes to help address unmet medical needs by finding new uses for previously-approved medications.
New uses for an already approved drug, be it a new indication or a new eligible patient population, would leverage existing safety data to accelerate the availability of treatments, the FDA explained in a May 11 release.
“Too many patients lack effective treatment options, even when promising science exists,” departing FDA Commissioner Marty Makary, M.D., explained in the announcement. “Drug repurposing can make better use of available scientific data to deliver effective treatment options for patients in need.”
To move the initiative along, the agency is asking patients, clinicians and other stakeholders to chime in on priority disease areas and potential drug candidates that could be repurposed. A public docket, published in the Federal Register on Tuesday, requests comment submissions by June 11 and specifically seeks suggestions regarding FDA-approved drugs for which “there appears to be no commercial interest in adding a new use” through a standard supplemental new drug application (sNDA).
The request is part of a “broader FDA initiative to update the labeling of FDA-approved drugs, when supported by sufficient evidence, to ensure that information in the labeling is clinically meaningful for health care providers and patients and scientifically up to date,” the agency noted in its release.
U.S. health officials have unleashed several moves recently that speak to the broader labeling efforts. Some related frameworks that the FDA points to in its docket include the oncology labeling-related Project Renewal and a September proposal from the Make America Healthy Again (MAHA) commission, which mentions repurposing drugs to treat chronic diseases.
One of the most prominent examples of the FDA’s relabeling of drugs outside of the standard approval pathway unfolded through Robert F. Kennedy’s push into the “autism epidemic” late last year. To propose a potential treatment for cerebral folate deficiency (CFD)’s “autistic features,” the FDA brought back to life GSK’s Wellcovorin (leucovorin calcium), a decades-old drug that GSK had stopped selling in the 1990s.
In October, the FDA reapproved the drug as it looked to position it as an autism treatment based on real-world findings. Five months later, the agency officially signed off on a label change that allows Wellcovorin to treat CFD in patients who have a certain confirmed gene variant. Instead of basing its approval on a clinical trial, the FDA relied on published literature and known data on the drug’s mechanism to make the call.
This approach is one of a few the FDA has used to support label updates for approved drugs, as explained in the docket.
Now, as the agency scouts for more approved drugs to make label changes to, it notes that drugs that could potentially meet the bar must already have data on their safety and effectiveness. However, the FDA welcomes suggestions for other drugs without such evidence, as they may guide “promising areas that warrant further study,” it explained.
As it stands, the FDA’s priority areas for the new initiative lay in metabolic diseases, neurodegenerative conditions, women’s and men’s health conditions, substance use disorders and rare diseases. Any input from its request for public comments will “help inform how the FDA refines its approach to evaluating and facilitating additional drug repurposing opportunities, including collaborations with federal partners such as the National Institutes of Health and the Centers for Medicare and Medicaid Services,” it said.
Although the Monday announcement was co-signed by Makary, the ex-commissioner will no longer have a hand in the initiative after his Tuesday resignation from the agency.
His departure follows a tumultuous 14 months in the top FDA spot, during which he drew criticism over several decisions including his stance and actions on the abortion pill mifepristone, fruit-flavored e-cigarettes, the Commissioner’s National Priority Review Voucher pilot program and more.