The US Food and Drug Administration (FDA) has accepted and granted priority review to Takeda’s biologics licence application (BLA) for its enzyme replacement therapy, TAK-755, to treat congenital thrombotic thrombocytopenic purpura (cTTP).
By replacing the missing or deficient ADAMTS13 enzyme, TAK-755 offers targeted therapy for addressing an unmet medical need for thrombotic thrombocytopenic purpura patients.
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If approved, the therapy is expected to become the first and only recombinant ADAMTS13 (rADAMTS13) replacement therapy to treat cTTP.
Takeda rare genetics and haematology therapeutic area unit head Daniel Curran said: “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.
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“We continue to be encouraged by the data and are working closely with the US FDA and other global regulatory bodies with the goal to bring this treatment to patients.”
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By GlobalDataThe BLA submitted to the FDA is supported by data obtained from the first Phase III randomised, controlled trial in cTTP patients, as well as the long-term data from a continuation study.
The Phase III trial assessed TAK-755’s clinical benefit across several clinically relevant endpoints, against plasma-based therapies, in a randomised cross-over study.
According to the interim results, TAK-755 reduced 60% incidence of thrombocytopenia events over plasma-based therapy.
The therapy previously received rare pediatric disease, fast track and orphan drug designation to treat cTTP from the FDA.
