Grandbrothers/iStock Editorial via Getty Images Ultragenyx ( NASDAQ: RARE ) said it would submit a Biologics License Application to the U.S. FDA seeking accelerated approval of UX111, a gene therapy for Sanfilippo syndrome, a rare metabolic disease.
The company noted that following a recent meeting with the agency, it was agreed that cerebral spinal fluid heparan sulfate could be used as a surrogate endpoint. A pre-BLA meeting with the agency is still needed, but Ultragenyx is eying a filing late this year or in early 2025. UX111 would be given as a one-time treatment.
Sanfilippo syndrome is a fatal lysosomal storage disease that mostly impacts the central nervous system. There are no approved therapies. Separately, after Wednesday's closing bell, the company said it has begun a public offering of up to $350M of common stock and pre-funded warrants, sending shares down ~3% in after-hours trading.
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