FDA Fast Tracks Recombinant ADAMTS13 Enzyme Replacement Therapy

Shire announced that the Food and Drug Administration (FDA) has granted Fast Track designation to SHP655 (historically BAX930) for the treatment of acute episodes of hereditary thrombotic thrombocytopenic purpura (hTTP) in patients with a constitutional ADAMTS13 deficiency.

The designation is based on preclinical data and results from a Phase 1 clinical trial for SHP655. The study showed that a single dose of SHP655 demonstrated comparable activity PK parameters to those estimated from fresh frozen plasma (FFP). Additionally, no serious adverse events were reported for SHP655. The company plans to initiate a randomized, open-label, 2-period crossover Phase 3 study for the treatment and prevention of acute TTP in patients with severe hereditary ADAMTS13 deficiency. 

SHP655 is a recombinant ADAMTS13, a von Willebrand factor-cleaving (VWF) protease. If approved, SHP655 will become the first ever recombinant ADAMTS13 enzyme replacement therapy.

For more information visit Shire.com.