FDA accepts Takeda's TAK-755 BLA for priority review

The US Food and Drug Administration (FDA) has accepted Japanese pharma major Takeda’s (TYO: 4502) Biologics License Application (BLA) for TAK-755, an enzyme replacement therapy for the treatment of congenital thrombotic thrombocytopenic purpura (cTTP), an ADAMTS13 deficiency disorder, for priority review.

The FDA has also granted TAK-755 Rare Pediatric Disease (RPD) designation for cTTP. TAK-755 has previously received Fast Track and Orphan Drug designation in cTTP.

If approved, TAK-755 would be the first and only recombinant ADAMTS13 (rADAMTS13) replacement therapy for cTTP, a disorder with considerable unmet patient need.