The FDA is looking to retool drugs with sufficient evidence to support their use in other indications, particularly those with unmet need.
The FDA is considering the “repurposing” of old drugs to expand their usage to other conditions, responding to medical needs that continue to be unaddressed.
On Monday, the regulator put out a call for input on the program, soliciting feedback from various stakeholders—including patients, doctors and researchers—regarding potential drugs worth revisiting and disease areas where drug repurposing could be useful. The agency is also looking for “innovative approaches” to identify candidates that can be retooled.
In recasting old products, the FDA will zero in on those for which “there are scientific data that could support approval of potential new uses but there appears to be limited commercial incentives to pursue approval of those uses,” according to a news release on Monday.
The program will initially address persistent medical need in the metabolic and neurodegenerative spaces, as well as rare diseases, substance use disorders and women’s and men’s health, though the FDA will remain open to other target areas that “stakeholders believe should be prioritized,” the announcement read.
“Too many patients lack effective treatment options, even when promising science exists,” FDA Commissioner Marty Makary said in a prepared statement. The FDA’s proposed program “can make better use of available scientific data to deliver effective treatment options,” he added.
Stakeholders will have 30 days from the publication of the Federal Register notice to submit their feedback, with the period for comment set to close June 11.
This new drug repurposing push comes as the agency is once again embroiled in controversy. On Friday afternoon, The Wall Street Journal reported that President Donald Trump has agreed to a plan to sack Makary, citing anonymous insiders with knowledge of the matter. Days earlier, one of Makary’s top deputies, Vinay Prasad, former director of the Center for Biologics Evaluation and Research, left the FDA after a similarly rocky tenure.
Makary and Prasad have overseen some contentious decisions at the agency, including the recent second rejection of Replimune’s melanoma therapy RP1. The denial has invited a deluge of criticism against the agency, with Makary earlier this month hitting back during an interview with CNBC, branding the stream of bad press against the FDA as “corporate spin.”
Makary’s termination remains unconfirmed. “I know nothing about it,” Trump told Fox News on Saturday. If it comes to pass, however, analysts expect Makary’s exit to be good for the industry, even though it will continue a pattern of leadership instability at the agency.
