Bayer’s regorafenib gains orphan status for GIST
pharmafile | February 4, 2011 | News story | Research and Development, Sales and Marketing | Bayer, FDA, GIST, gastrointestinal stromal tumours, orphan drug, regorafenib
Bayer’s investigational compound regorafenib has been granted orphan drug status for the treatment of patients with gastrointestinal stromal tumours (GIST) by FDA.
“This is an important step in the overall development process for this investigational compound,” said Kemal Malik, MD, head of global development and member of the Bayer healthcare executive committee.
The decision brings regorafenib one step closer to competing with the two major drugs already licensed to treat GIST – now a well established market, with Novartis’ Glivec and Pfizer’s Sutent leading the field.
A phase III study of the drug has just began enrolling patients with metastatic and/or unresectable gastrointestinal stromal tumours (GIST) whose disease has progressed despite prior treatment with at least Glivec and Sutent.
The trial is estimated to enrol 170 patients, who will be randomised in a 2:1 ratio to receive either regorafenib or placebo. Subjects receiving placebo who experience disease progression may be offered open-label regorafenib treatment (cross-over option).
The primary endpoint of this trial is progression-free survival (PFS), and secondary endpoints include overall survival, time to progression, disease control rate (DCR), tumour response rate, duration of response, and safety. All patients will enter the Survival Follow-Up Period upon discontinuation of study treatment, during which assessment of survival status will be performed every three months.
Andrew McConaghie
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