08:38 AM EST, 02/18/2025 (MT Newswires) -- Ultragenyx Pharmaceutical ( RARE ) said Tuesday that its UX111, ABO-102, AAV gene therapy, a treatment for patients with Sanfilippo syndrome type A, has been accepted for review by the U.S. Food and Drug Administration.

In response to the company's request for accelerated approval, the U.S. health regulator has given a decision date of Aug. 18. The agency also said it is not planning to hold an advisory committee meeting to discuss the application.

UX111 is a vivo gene therapy for Sanfilippo syndrome type A, a rare fatal lysosomal storage disease with no approved treatment that mainly impacts the brain.

Ultragenyx said that the latest studies demonstrated that treatment with UX111 resulted in a statistically significant improvement in the cognition, receptive communication, and expressive communication of treated patients compared to untreated patients.