What is happening?
A promising gene therapy for Sanfilippo syndrome—a relentless disease that robs children of speech, mobility, and independence—is stalled by FDA delays. Trials show UX111 can slow or preserve these abilities, offering hope where no treatments exist.
Yet despite strong data, approval was rejected over minor manufacturing issues, delaying access for at least 6–12 months. For children with Sanfilippo, every month means irreversible loss.
Our kids are losing their voices, so we need yours to help by contacting HHS Secretary Kennedy and FDA Commissioner Makary and ensure:
Swift review of resubmissions for rare disease drugs, especially for Sanfilippo and Barth.
Engagement with sponsors to resolve minor issues before rejecting applications.
The FDA holds direct meetings with patient communities impacted by recent delays, including Sanfilippo and Barth.
The FDA makes full use of existing regulatory flexibility when the science is strong and safety risks are minimal.
