Market PotentialTA-ERT has Fast Track designation, Rare Pediatric Disease Designation (RPDD), and Orphan Drug status in the U.S. and EU.
Regulatory ProgressThe FDA confirmed that the completed clinical and non-clinical studies of TA-ERT were sufficient for a BLA submission and provided confirmatory trial design guidance.
Strategic AcquisitionsSpruce Biosciences acquired tralesinidase alfa, an enzyme replacement therapy, originally developed by BioMarin, which is in late-stage development and has a seemingly de-risked path to approval.