Shots:
- Ultragenyx has completed rolling submission of a BLA to the FDA seeking approval of DTX401 (pariglasgene brecaparvovec) for the treatment of GSDIa
- BLA is supported by data from 52 pts with 6yrs. of follow-up. Previously reported P-III (GlucoGene) trial results showed significant reductions in both quantity & frequency of daily cornstarch intake, improved euglycemia levels, while maintaining low levels of hypoglycemia, with meaningful QoL gains per PGIC
- The company was granted rolling review & submitted the non-clinical & clinical modules in Aug 2025, plus has now completed the BLA package with submission of the CMC module
Ref: Ultragenyx |  Image: Ultragenyx  | Press Release
Related News: Ultragenyx Completes Patient Enrolment in P-III (Aspire) Trial of GTX-102 for Angelman Syndrome
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