NasdaqGS:RAREBiotechs
Ultragenyx Pharmaceutical (RARE): Valuation in Focus After Launching FDA Submission for DTX401 Gene Therapy
If you have been tracking Ultragenyx Pharmaceutical (RARE), the latest regulatory move may have caught your eye. The company just kicked off a rolling submission for its Biologics License Application to the FDA, targeting approval for DTX401, an AAV gene therapy designed for Glycogen Storage Disease Type Ia. This step is backed by data from its Phase 3 trial, with encouraging reductions in patient daily cornstarch needs. It is also supported by several FDA designations that signal a real...