10:36 AM EST, 12/19/2024 (MT Newswires) -- Ultragenyx Pharmaceutical ( RARE ) said Thursday it submitted a biologics license application to the US Food and Drug Administration for accelerated approval for UX111 to treat Sanfilippo syndrome type A, a lysosomal storage disease that mainly affects the brain.

The company said the submission is supported by available data, including from a study that showed "rapid and sustained decreased levels" of heparan sulfate in cerebral spinal fluid in patients treated with UX111.

Ultragenyx shares were down 1.9% in recent trading.

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