Ultragenyx FDA OKs Resubmission for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome

 If approved, UX111 will be the first approved therapy for the treatment of Sanfilippo syndrome Type A, a rare disease affecting young children that leads to progressive, irreversible neurodegeneration and early death

PDUFA action date set for September 19, 2026

https://finviz.com/news/342120/ultragenyx-announces-us-fda-acceptance-of-bla-resubmission-for-ux111-aav-gene-therapy-to-treat-sanfilippo-syndrome-type-a-mps-iiia