FDA accepts BLA for TAK 755 an enzyme replacement therapy for the treatment of congenital thrombotic thrombocytopenic purpura.- Takeda

The FDA has also granted TAK 755 Rare Pediatric Disease (RPD) designation for cTTP. TAK 755 has previously received Fast Track Designation 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.

cTTP is an ultra-rare inherited form of thrombotic thrombocytopenic purpura (TTP), a chronic and debilitating clotting disorder caused by a deficiency in ADAMTS13 protease. Acute TTP has a mortality rate of greater than 90%, if left untreated.

The BLA is supported by the totality of the evidence provided by efficacy, pharmacokinetic, safety and tolerability data from the first randomized, controlled trial in cTTP, and supported by long-term safety and efficacy data from a continuation study. The Phase III trial was designed to evaluate the clinical benefit of TAK 755 across multiple clinically relevant endpoints, compared to plasma-based therapies, in a randomized cross-over study. The interim results, announced by Takeda in January 2023, showed that TAK 755 reduced the incidence of thrombocytopenia events by 60% (95% Confidence Interval, 30%-70%), an important marker of disease activity in cTTP, as compared to plasma-based therapy. The proportion of subjects experiencing adverse events determined to be related to the treatment was substantially lower among subjects during treatment with TAK 755 (8.9%) compared to plasma-based therapies (47.7%). The interim analysis of the Phase III results will be presented at an upcoming scientific meeting. Takeda is also investigating the safety, efficacy and pharmacokinetics of TAK-755 treatment in immune-mediated TTP (iTTP) in an ongoing Phase IIb study.