Posted by Michael Wonder on 01 Feb 2026
Ultragenyx resubmits biologics license application for UX111 AAV gene therapy to treat Sanfilippo syndrome type A (MPS IIIA) to US FDA
30 January 2026 - Company expects up to six month review period per FDA guidelines.
Ultragenyx today announced that it has resubmitted its biologics license application seeking accelerated approval for UX111 (rebisufligene etisparvovec) AAV9 gene therapy as a treatment for patients with Sanfilippo syndrome type A to the US FDA.
