Regorafenib Granted Orphan Drug Status

9 February 2011 (Last Updated February 9th, 2011 18:30)

Bayer HealthCare investigational anti-cancer compound regorafenib has been granted orphan drug status by the US Food and Drug Administration (FDA). The status has been granted for the treatment of patients with gastrointestinal stromal tumours (GIST). Bayer HealthCare manag

Bayer HealthCare investigational anti-cancer compound regorafenib has been granted orphan drug status by the US Food and Drug Administration (FDA).

The status has been granted for the treatment of patients with gastrointestinal stromal tumours (GIST).

Bayer HealthCare managing director Kemal Malik said, "This is an important step in the overall development process for this investigational compound."

In January, the company announced that it had begun to enrol patients in an international Phase III trial to evaluate the compound for the treatment of patients with metastatic or unresectable GIST, whose disease had progressed despite prior treatment.

The trial is expected to enrol 170 patients, with a primary endpoint of progression-free survival and secondary endpoints including overall survival, time to progression and disease control rate.