FDA grants Priority Review for Takeda’s cTTP treatment
James Spargo | May 17, 2023 | News story | Research and Development | Biologics License Application, Cardiology, FDA, Takeda, cTTP
Japanese biopharmaceutical company Takeda has announced that the US Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) and granted Priority Review for Takeda’s TAK-755, for the treatment of congenital thrombotic thrombocytopenic purpura (cTTP).
cTTP is an ultra-rare inherited form of TTP, a chronic and debilitating clotting disorder. Acute TTP has a mortality rate of >90% if left untreated.
TAK-755 is an enzyme replacement therapy and the first recombinant ADAMTS13 protein in development. It works by replacing the missing or deficient ADAMTS13 enzyme in cTTP patients.
The BLA was awarded based on evidence from a phase 3 randomised, controlled study which highlighted the safety, efficacy, pharmacokinetic and tolerability of TAK-755. In January 2023, Takeda announced interim results which showed TAK-755 reduced the incidence of thrombocytopenia events by 60% compared to plasma-based therapy. The number of participants who experienced adverse effects was also lower ‒ 8.9% during treatment with TAK-755, compared to 47.7% during treatment with plasma-based therapies.
Daniel Curran MD, head of Rare Genetics and Haematology Therapeutic Area Unit at Takeda, stated: “There is a critical need for treatment options for people living with cTTP, an ultra-rare, life-threatening disorder that has no therapies specifically approved for prophylactic treatment. TAK-755 is the first and only treatment in clinical development that provides targeted replacement of ADAMTS13, addressing the underlying cause of the disease. We continue to be encouraged by the data and are working closely with the FDA and other global regulatory bodies with the goal to bring this treatment to patients.”
James Spargo
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