In 2015, the United States Food and Drug Administration (US FDA) and other Department of Health and Human Services agencies helped respond to a public health emergency concerning Zika Virus during which 5,600 cases of the virus were reported in the United States mainland and 37,000 cases in Puerto Rico and other US Territories.
Every organization goes through ups and downs. And the Food and Drug Administration is no different.
But the stakes are higher when you’re tasked with protecting public health. So, it’s understandable when “Put the F Back in FDA” started trending online in 2022 as a way to knock the agency for the perception that it focused too much on things that were not its Human Foods Program, such as drugs.
When the U.S. Food and Drug Administration made the opioid overdose reversal drug naloxone an over-the-counter medication last summer, health care advocates praised the removal of the prescription barrier as an effective tool to prevent fentanyl deaths.
At Denali Therapeutics, where I serve as chief medical officer and head of development, we are eager to develop new treatments for progressive and debilitating neuronopathic mucopolysaccharidoses diseases with our novel technology. We are not alone—several other biopharma companies also have drug candidates that have shown promise in clinical testing. Unfortunately, as so many families and physicians know, the road to approval of new treatments for rare diseases is long and slow.
A meeting convened by the Reagan-Udall Foundation for the FDA on qualifying rare disease biomarkers for accelerated approvals could be an inflection point for patients with ultrarare diseases, their families, biotech companies, and FDA.
The FDA will increasingly use accelerated approval for gene therapies aimed at rare conditions, according to Peter Marks, the FDA’s top official overseeing cell and gene therapies, the clearest signal yet of how widely the agency will deploy its expedited pathway.
On February 21, the Biotechnology Innovation Organization’s (BIO) soon-to-be CEO, John F. Crowley, joined an event about biomarkers and rare disease regulatory pathways, hosted by the Reagan-Udall Foundation for the Food and Drug Administration in Washington, D.C.
Flexible thinking and rigorous standards will both be needed to develop psychedelics as drug therapies in order to surmount the many complicating factors, from unique ‘set and setting’ aspects to functional unblinding, speakers at Reagan-Udall Foundation meeting agree.
Even as FDA stands by its decision that CBD is not safe for use in foods, beverages and dietary supplements, the agency is considering how best to ensure it is clearly labeled, safely manufactured and consumers understand what it is, according to a senior agency official.
Officials with the Food and Drug Administration (FDA) recently joined scientists at a public meeting on next steps for conducting research to develop psychedelic medicines.
