“The FDA’s New Leader Must Keep the Agency’s Promises to Rare Disease Patients”
Elise Drake-Esposito, PhD, is a Research Project Coordinator for Cure Sanfilippo Foundation. She is also mother to Keira, her 16-year-old daughter who has Sanfilippo Syndrome, and she urges whoever becomes the new Commissioner of the U.S. Food & Drug Administration (FDA) to act with flexibility and urgency in serious and life-threatening conditions when it comes to rare diseases in a May 27, 2026 op-ed in the DC Journal.
In the op-ed, Esposito points out that expedited attention has been granted for certain programs and drugs and notes that “rare disease patients deserve that same level of urgency, especially when there are no approved alternatives and the consequences of delay are irreversible.”
“One of the most painful things about this disease is knowing that promising treatments are emerging, but funding barriers and manufacturing and procedural slow-downs continue to derail progress. UX111 is a life-changing gene therapy. Clinical trial results show that a single dose of UX111 gene therapy can reduce toxin buildup for more than eight years, and when administered early in life, it can slow cognitive decline. Even when administered at later ages, it has a significant effect in delaying disease progression. Unfortunately, children who have not been able to access this in a clinical trial have continued to suffer losses while waiting for access and approval.”
Currently, there are no approved therapies for Sanfilippo Syndrome. FDA is currently reviewing a application for UX111, a gene therapy that has been undergoing regulatory review, off and on, since last year.
There are concerns about how the extensive leadership changes underway at FDA may be impacting the agency’s expediency and focus. As Esposito says, diseases like Sanfilippo Syndrome do not pause while systems lag; these children can’t afford to wait.