NasdaqGS:RAREBiotechs
Ultragenyx Pharmaceutical (RARE) Is Up 12.7% After FDA Grants Priority Review To DTX401 Gene Therapy - Has The Bull Case Changed?
Ultragenyx Pharmaceutical recently announced that the FDA accepted for review, with Priority Review, its Biologics License Application for DTX401 gene therapy to treat Glycogen Storage Disease Type Ia, supported by data from 52 patients and up to six years of follow-up.
This move positions Ultragenyx to potentially introduce the first pharmacologic therapy targeting the underlying cause of GSDIa, leveraging its new U.S.-based gene therapy manufacturing facility and multiple regulatory...