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news.Bayer HealthCare Pharmaceuticals has received orphan drug designation from the US Food and Drug Administration (FDA) for its investigational compound regorafenib (BAY 73-4506), for the treatment of patients with gastrointestinal stromal tumours (GIST).
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Bayer had initiated patient enrolment for a randomised, double-blind, placebo-controlled Phase III study of regorafenib plus best supportive care versus placebo plus best supportive care for subjects with metastatic and/or unresectable GIST.
The trial is estimated to enroll 170 patients, who will be randomised in a 2:1 ratio to be treated with either regorafenib or placebo.
The primary endpoint of the trial is progression-free survival (PFS), and secondary endpoints will be overall survival (OS), time to progression (TTP), disease control rate (DCR), tumour response rate (RR), duration of response (DOR), and safety.
The orphan drug designation is granted by the FDA to those drugs that treat medical condition affecting fewer than 200,000 people in the US.