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.
Price: 42.30, Change: -0.64, Percent Change: -1.49
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