“Right now, 30 million Americans – half of them children – have rare diseases, 95% of which have no approved treatments. That’s why it’s so concerning that progress has been blocked by the recent rash of FDA denials of proposed treatments from drugmakers, despite the benefit and safety these treatments are showing in clinical trial patients,” stated Dr. Cara O’Neill, Chief Science Officer & Co-Founder of Cure Sanfilippo Foundation in a Sept. 1, 2025, op-ed in USA Today.
Earlier this year, FDA issued Complete Response Letters rejecting applications for Ultragenyx’s Sanfilippo Syndrome A gene therapy (UX111), Capricor’s cell therapy for Duchenne Muscular Dystrophy, Replimune’s biologic for advanced melanoma, and Stealth’s drug for Barth syndrome.
“With each delay, the FDA timeclock gets reset. That means months applying for meetings and preparing extensive documents, followed by an FDA review process that normally takes at least six months – that may still result in more delays or denials. However, for patients whose diseases can go from “bad” to “irreversible decline” in the span of weeks, this means many children will die or suffer severe permanent disability waiting for help,” explains O’Neill.
“My family has seen firsthand what happens when access timelines and the progressive nature of a disease don’t align … With no approved treatments, families are forced to watch their children suffer over a decade or more developing seizures and pain while losing the ability to walk, to communicate, to eat on their own or even sleep at night,” states O’Neill.
Dr. O’Neill urges FDA to “move at the speed of patient need.” She applauds signals from FDA Commissioner Marty Makary to increase transparency and support rapid advancement of treatments for rare and serious diseases, but voices concern that those words have to be matched by an FDA commitment to patient-focused drug development.
Read the complete USA Today op-ed by Dr. O’Neill.