Bristol Myers Squibb Voluntarily Withdraws U.S. Indication for Opdivo Monotherapy in Sorafenib-Previously Treated Hepatocellular Carcinoma

By | Shibei

On July 23, Bristol-Myers Squibb (BMS) announced that, following consultations with the U.S. FDA, it is voluntarily withdrawing the indication in the U.S. market for Opdivo (nivolumab) monotherapy for patients with hepatocellular carcinoma (HCC) who have previously been treated with sorafenib. This decision by BMS is based on the April meeting of the Oncologic Drugs Advisory Committee and subsequent discussions with the FDA.

This indication for Opdivo was first granted accelerated approval by the FDA in 2017, making it the first immunotherapy approved for this patient population. The accelerated approval was based on tumor response data from the Phase I/II CheckMate-040 clinical study. However, in the confirmatory clinical trial CheckMate-459, a head-to-head comparison of Opdivo versus sorafenib as first-line treatment for patients with hepatocellular carcinoma, the primary endpoint of overall survival did not achieve statistical significance in the prespecified analysis.

BMS stated that, following the withdrawal of the monotherapy indication, the Opdivo and Yervoy combination therapy remains approved for patients with hepatocellular carcinoma (HCC) who have progressed on or are intolerant to sorafenib.

Liver cancer is the fourth leading cause of cancer death worldwide, and hepatocellular carcinoma (HCC) is the most common type of liver cancer, as well as the fastest-growing cause of cancer-related mortality in the United States. HCC is typically diagnosed at an advanced stage, when effective treatment options are limited, with a 5-year survival rate of less than 15% under the first-line standard of care. Although most HCC cases are caused by hepatitis B virus (HBV) or hepatitis C virus (HCV) infection, the rising prevalence of metabolic syndrome and non-alcoholic steatohepatitis (NASH) is expected to drive an increase in liver cancer incidence.