Shire’s drug picked up from Baxalta given fast track designation

pharmafile | March 23, 2017 | News story | Research and Development, Sales and Marketing Shire, rare diseases 

Shire has announced that its drug, SHP655, has received a fast track designation based on pre-clinical trials and positive results from a Phase 1 study. The drug is a treatment for patients with hereditary thrombotic thrombocytopenic purpura (hTTP), a rare blood disorder 3,000 to 4,000 patients suffer from globally.

hTTP is a disorder in which individuals are congenitally deficient in ADAMTS13, an enzyme that cleaves the von Willebrand factor protein involved in blood clotting. The risk of death is high if the condition is left untreated.

The advantages of Shire’s potential treatment are that it would offer an alternative to standard therapy, wherein fresh plasma is administered to patients but can contain variable amounts of ADAMTS13. Shire’s treatment would enter the market, if approved, as the first recombinant ADAMTS13 enzyme replacement therapy.

The drug was formerly known as BAX930 and was acquired as part of Shire’s mega-merger with Baxalta. Phase 1 results were posted by Baxalta back in May of 2016, revealing that 15 patients who were treated during the trial had suffered only minor adverse events that were resolved without the need of medication.

“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,” said Philip J. Vickers, Head of R&D, Shire. “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 next step will be a Phase 3 trial for the drug, which is already planned as a randomized, open-label, 2-period crossover study with a single arm continuation to evaluate the safety and efficacy of SHP655. The study will take place in the US, Europe and Japan.

Ben Hargreaves

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