Supplemental approval of Opdivo for expanded use for treatment of cancer of unknown primary in Japan.

The supplemental approval in Japan is based on the result from an investigator-initiated clinical study (NivoCUP study), conducted under the initiative of Kindai University Hospital, evaluating Opdivo in patients with cancer of unknown primary (CUP).

This approval is based on the result from an investigator-initiated clinical study (NivoCUP study), conducted under the initiative of Kindai University Hospital, evaluating Opdivo in patients with cancer of unknown primary (CUP). In this study, the observed response rate (ORR) in patients previously treated with chemotherapy (central assessment), the primary endpoint, was 22.2% (95% confidence interval: 11.2 - 37.1). Since the lower limit of the confidence interval exceeded a preset threshold ORR of 5%, the study met the primary endpoint. Furthermore, ORR in the entire patient population regardless of treatment history (central assessment) was 21.4% (95% confidence interval: 11.6 - 34.4), indicating the antitumor effect of Opdivo across treatment history.

Cancer of the Uknown Primary is defined as a malignant tumor of which primary lesion is unknown despite thorough search and is histologically proven to be a metastatic lesion. CUP is a pathological condition that has already advanced or metastasized at the time of diagnosis, and more than half of patients have metastasis to multiple organs. CUP is a serious and life-threatening disease having extremely poor prognosis with a median survival of 6 - 9 months.