04:50 PM EDT, 06/12/2024 (MT Newswires) -- Ultragenyx Pharmaceutical ( RARE ) said Wednesday it intends to file late this year or early next year a biologics license application with the US Food and Drug Administration for the accelerated approval of its UX111 novel gene therapy for Sanfilippo syndrome.

The Sanfilippo syndrome is a rare, fatal lysosomal storage disease that primarily affects the central nervous system.

The BLA filing will be based on the available data from the ongoing pivotal Transpher A study evaluating the safety and efficacy of UX111 in children, it said.

Shares of the company fell about 3% in recent Wednesday after-hours activity.

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