Ultragenyx Pharmaceutical Inc. (RARE) announced that it has completed the rolling submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration for DTX401 AAV gene therapy (pariglasgene brecaparvovec), intended as a treatment for Glycogen Storage Disease Type Ia (GSDIa).
If approved, DTX401 would become the first therapy to directly address the underlying cause of GSDIa, marking a significant milestone for patients living with this rare genetic disorder.
The company had previously submitted the non-clinical and clinical modules to the FDA in August and has now finalized the application package with the submission of the chemistry, manufacturing, and controls (CMC) module.
RARE closed Tuesday's regular trading on the NasdaqGS at $22.78, up $3.06 or 15.52%. In overnight trading at 8:46 PM EST, the stock eased slightly to $22.41, down $0.37 or 1.62%.
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