Ultragenyx Pharmaceutical Inc. (RARE), announced on Thursday that it has submitted a Biologics License Application to the U.S. FDA for UX111, an AAV gene therapy for Sanfilippo Syndrome Type A.
Sanfilippo Syndrome Type A, or MPS IIIA, is a rare, fatal neurodegenerative disease in which the body is unable to break down a complex sugar molecule called heparan sulfate. This disorder primarily affects children, and at present, no approved treatments are available.
The BLA submission was supported by data from an ongoing pivotal Transpher A study, which demonstrated that treatment with UX111 resulted in rapid and in sustained reduction in heparan sulfate (HS) levels in cerebrospinal fluid (CSF), a key biomarker of the disease, in patients with Sanfilippo syndrome type A.
This reduction in HS was linked to improved long-term cognitive development compared to the natural history of the disease, offering hope for better outcomes for patients, according to the company.
UX111 has received several regulatory designations, including Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Rare Pediatric Disease, and Orphan Drug designations in the U.S., as well as PRIME and Orphan Medicinal Product designations in the EU.
If approved, UX111 would be the first approved therapy in the U.S. for Sanfilippo Syndrome Type A.
RARE closed Wednesday's (Dec.18.2024) trading at $42.94, down 4.47%. In premarket trading, the stock is down by another 1.47% at $42.31.
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