FDA Approves Opdivo for Liver Cancer Previously Treated with Sorafenib

In trial, 14.3% of patients responded to the drug

The FDA has approved nivolumab injection (Opdivo, Bristol-Myers Squibb) for intravenous use for the treatment of patients with hepatocellular carcinoma (HCC) who have been treated previously with sorafenib (Nexavar, Bayer HealthCare).

Accelerated approval for this indication was granted based on tumor response rate and durability of response. In the CheckMate-040 trial, 14.3% of patients responded to treatment with nivolumab. The percentage of patients with a complete response was 1.9% (three out of 154) and the percentage of patients with a partial response was 12.3% (19 out of 154). Among responders ( n= 22), responses ranged from 3.2 to 38.2-plus months; 91% of those patients had responses of six months or longer and 55% had responses of 12 months or longer.

The burden of liver cancer in the U.S. is significant and is expected to increase in the decades to come. A recently-released American Cancer Society (ACS) report published in CA: A Cancer Journal for Clinicians notes that death rates for liver cancer are increasing at a faster pace than any other cancer, doubling since the mid-1980s.

“Unfortunately, the majority of HCC patients are diagnosed with advanced-stage disease and are not candidates for potentially curative surgical interventions,” said Adrian M. Di Bisceglie, MD, co-director of the Saint Louis University Liver Center and Chief of Hepatology. “More options are needed for advanced-stage HCC patients who have failed prior systemic therapy.”

At the advanced stage, treatment options for HCC are limited and there is a high unmet need for patients who are intolerant to or who have progressed on sorafenib therapy.

CheckMate-040 included a phase 1/2, open-label, multicenter study evaluating nivolumab in patients with HCC who progressed on or were intolerant to sorafenib. In this study, 154 patients received nivolumab 3 mg/kg administered intravenously every two weeks. The recommended dose is 240 mg administered as an intravenous infusion over 60 minutes every two weeks until disease progression or unacceptable toxicity.

Efficacy outcome measures included confirmed overall response rate (as assessed by blinded independent central review using RECIST v1.1 and modified RECIST for HCC) and duration of response. The median age of patients participating in the study was 63 (range: 19-81), all patients had received prior sorafenib therapy, and 19% of patients had received two or more prior systemic therapies. Patients were enrolled regardless of PD-L1 expression level and whether or not they were infected with active hepatitis B  or hepatitis C virus.

Source: Bristol-Myers Squibb; September 22, 2017.