FDA fast-tracks Shire's SHP655 for patients with ADAMTS13

Shire PLC's recombinant ADAMTS13 drug, SHP655, has been given fast-track designation by the U.S. Food and Drug Administration.
SHP655 is used in the treatment of acute episode of hereditary thrombotic thrombocytopenic purpura (hTTP) in patients with a constitutional deficiency of the von Willebrand factor-cleaving (VWF) protease ADAMTS13.
"As the leader in rare disease, Shire is committed to providing an innovative pipeline of world class therapeutics to the patients that need them most,” Philip Vickers, head of R&D at Shire, said in a statement. “Today’s confirmation from FDA that SHP655 for hereditary thrombotic thrombocytopenic purpura has been granted Fast Track designation reaffirms the significant unmet need that exists for this patient population and provides hope of reducing morbidity in patients with hTTP.”
The FDA’s fast-track designation was given after a Phase 1 study; the designation will help facilitate the development and review of the drug to fill an unmet need in treating serious conditions. Shire noted that this does not guarantee FDA approval and the company is moving forward with its Phase 3 trial, which will include a randomized, open-label, two-period crossover study with a single arm continuation to evaluate the safety and efficacy of SHP655.