Bristol Myers Scores FDA Approval for Opdivo-Yervoy Combination as First-Line HCC Therapy

Bristol Myers Squibb announced that the U.S. Food and Drug Administration (FDA) has approved Opdivo (nivolumab) in combination with Yervoy (ipilimumab). The approval applies to the first-line treatment of adults with unresectable or metastatic hepatocellular carcinoma (HCC), the most common type of primary liver cancer. This decision is based on findings from the global Phase 3 CheckMate-9DW trial. 

Phase 3 Trial Enrolled 668 Patients to Compare Opdivo-Yervoy Combo with Standard Treatments in Advanced HCC

The FDA’s approval is supported by data from the global Phase 3 CheckMate-9DW trial, a randomized, open-label study. The trial assessed Opdivo combined with Yervoy against an investigator-selected tyrosine kinase inhibitor monotherapy, either lenvatinib or sorafenib, in patients with unresectable or metastatic HCC who had not received prior systemic therapy. The combination treatment showed a statistically significant improvement in overall survival (OS) and overall response rate (ORR) compared to the monotherapy group. It remains the only study to demonstrate superior outcomes over this comparator regimen in support of an FDA approval.

The trial enrolled a total of 668 patients. Patients in the Opdivo-Yervoy group received intravenous infusions every three weeks for up to four doses. After that, they continued with Opdivo alone every four weeks for up to two years, or until the disease progressed or side effects became too severe.

Patients in the control group took either lenvatinib once a day (with the dose based on body weight) or sorafenib twice a day by mouth. Researchers set overall survival as the primary goal of the study. They also measured how many patients responded to treatment and how long it took for symptoms to worsen. The study did not aim to directly compare the Opdivo-Yervoy combination to each individual drug in the control group.

“The CheckMate-9DW approval is an important advancement for patients, considering the incidence of liver cancer has tripled in the last four decades, yet prognosis for HCC patients remains poor,” said Aiwu Ruth He, MD, PhD, a CheckMate-9DW study investigator while at MedStar Georgetown University Hospital.

HCC Cases in the U.S. Projected to Reach 42,240 in 2025 with 30,090 Deaths

HCC is the most common type of primary liver cancer in adults. In the United States, liver cancer ranks as the sixth leading cause of cancer-related deaths. HCC is often diagnosed at an advanced stage and is typically linked to a poor prognosis due to limited effective treatment options.

In 2025, an estimated 42,240 people in the U.S. are expected to be diagnosed with liver cancer, and approximately 30,090 are projected to die from the disease. Since 1980, the incidence of liver cancer in the U.S. has tripled, and mortality rates have doubled. HCC commonly occurs in individuals with chronic hepatitis B or C infections or liver cirrhosis. However, rising rates of metabolic syndrome and metabolic dysfunction-associated steatohepatitis are also expected to contribute to increasing cases of HCC.

In 2020, the U.S. FDA granted accelerated approval to the Opdivo and Yervoy combination based on data from the Phase 1/2 CheckMate-040 trial. This approval established the regimen as a second-line treatment for patients with advanced HCC who had previously received sorafenib. The FDA’s latest decision converts the accelerated approval to full approval and expands the use of the combination to the first-line setting. 

“Bringing Opdivo plus Yervoy to patients with HCC in the first-line setting is a testament to our ongoing commitment to research and delivering important progress for people living with cancer,” said Wendy Short Bartie, senior vice president of Oncology Commercialization at Bristol Myers Squibb. “Today’s approval builds on the legacy of our dual immunotherapy and the value it has brought to patients for years.”

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Denisse Sandoval