sanfilippo
2 stories tagged "sanfilippo"
Drugs & FDA
The FDA Approved a $3.95 Million Gene Therapy It Rejected Fourteen Months Ago
In July 2025 the FDA refused to approve this Sanfilippo gene therapy and faulted the factory, not the science. Fourteen months later it is the first treatment for a fatal childhood brain disease, cleared on 17 treated children against a comparison group the maker built, at $3.95 million a dose.
Drugs & FDAThe Data on the First Sanfilippo Gene Therapy Were Ready Last Summer. The FDA Spent the Next Year on the Factory.
The FDA said Ultragenyx's clinical data were robust in July 2025, then rejected its Sanfilippo gene therapy over manufacturing questions. Approval came fourteen months later, for children who lose skills and die on a clock.