Ultragenyx Pharmaceutical Inc (RARE) today announced that the FDA has accepted for priority review its Biologics License Application for UX11, an AAV gene therapy proposed for the treatment of Sanfilippo syndrome type A.
The U.S. regulatory agency has set the decision date for UX111 as August 18, 2025.
Sanfilippo Syndrome Type A, or MPS IIIA, primarily affects children and results in the body's inability to break down heparan sulfate, a complex sugar molecule. This disease leads to progressive neurodegeneration, cognitive decline, and early death, with no approved treatments available.
The BLA submission was based on data from an ongoing pivotal trial, dubbed Transpher A study, which demonstrated that UX111 significantly reduced levels of heparan sulfate or HS in the cerebrospinal fluid or CSF of patients.
The reduction of heparan sulfate was associated with substantial improvements in long-term cognitive function, compared to untreated patients, suggesting that UX111 may offer new hope for better outcomes in MPS IIIA patients.
UX111 has received Regenerative Medicine Advanced Therapy or RMAT, Fast Track, Rare Pediatric Disease, and Orphan Drug designations in the U.S. It also holds PRIME and Orphan Medicinal Product designations in the EU.
If approved, UX111 would become the first-ever approved therapy for Sanfilippo Syndrome Type A in the U.S.
RARE closed Friday's (Feb.14 2025) trading at $42.49 down by 2.37%. In premarket trading Tuesday the stock is down by 1.41% at $43.09.
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