Ultragenyx Pharmaceutical has announced that the FDA has accepted its Biologics License Application (BLA) for the gene therapy UX111, which is designed to treat Sanfilippo Syndrome.
This acceptance marks a significant step forward for Ultragenyx as it seeks to bring this treatment to patients affected by this rare genetic disorder.
The company is now poised to advance its efforts in addressing the needs of individuals with Sanfilippo Syndrome, pending further regulatory developments.