Recombinant ADAMTS13 Replacement Therapy Under Review for Congenital TTP

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Congenital thrombotic thrombocytopenic purpura is an ADAMTS13 deficiency disorder.

The Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) for TAK-755 for the treatment of congenital thrombotic thrombocytopenic purpura, an ultra-rare, chronic, clotting disorder in which blood clots form in the small blood vessels.

Congenital thrombotic thrombocytopenic purpura (cTTP) results from a deficiency in ADAMTS13, a von Willebrand factor (VWF) cleaving protease. This leads to an accumulation of ultra-large VWF multimers in the blood and uncontrolled platelet aggregation and adhesion. TAK-755 is a recombinant ADAMTS13 protein designed to replace the missing or deficient ADAMTS13 enzyme.

The BLA is supported by data from a randomized, controlled phase 3 study ( Identifier: NCT03393975). Patients were randomly assigned to receive either TAK-755 or the current standard of care (SOC) therapy (eg, plasma-based therapies).

Interim results showed that the incidence of thrombocytopenia events was reduced by 60% (95% CI, 30-70) with TAK-755 compared with SOC. Moreover, a significantly lower proportion of patients experienced adverse events during treatment with TAK-755 (8.9%) vs SOC (47.7%).

Long-term safety and efficacy data from a continuation study ( Identifier: NCT04683003) were also included in the application.

“TAK-755 is the first and only treatment in clinical development that provides targeted replacement of ADAMTS13, addressing the underlying cause of the disease,” said Daniel Curran, MD, Head, Rare Genetics & Hematology Therapeutic Area Unit at Takeda. “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.”

TAK-755 has been granted Fast Track, Orphan Drug, and Rare Pediatric Disease designations for this indication.