FDA Approves Treatment for Unresectable or Metastatic Hepatocellular Carcinoma

Science laboratory test tubes, laboratory equipment | Image Credit: © BillionPhotos.com - © BillionPhotos.com - stock.adobe.com

Bristol Myers Squibb (BMS) announced on April 11, 2025 that FDA has approved Opdivo (nivolumab) plus Yervoy (ipilimumab) as a first-line treatment for unresectable or metastatic hepatocellular carcinoma (HCC). According to BMS, HCC is the most common primary liver cancer.

The approval was based on results from the global Phase III randomized, open-label CheckMate-9DW trial. The trial evaluated the use of nivolumab plus ipilimumab against tyrosine kinase inhibitor monotherapy (lenvatinib or sorafenib) in patients with unresectable or metastatic HCC who have not received prior systemic therapy. According to BMS, the combination of nivolumab plus ipilimumab showed significant overall survival and overall response rate versus the comparator arm.

“In the CheckMate-9DW trial, in which 85% of patients in the comparator arm were treated with lenvatinib and 15% were treated with sorafenib, mOS with Opdivo plus Yervoy (n=335) was 23.7 months (95% CI: 18.8-29.4) vs. 20.6 months (95% CI: 17.5-22.5) with lenvatinib or sorafenib (n=333; HR=0.79; 95% CI: 0.65-0.96 P=0.0180), reducing the risk of death by 21%,” BMS explained in the press release (1).

“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, in the press release. “The availability of a new first-line treatment option that demonstrated a deep response can offer adults with this form of liver cancer long-term overall survival and may help address an unmet need. Given the strength of evidence from the trial, especially considering the selection and performance of a strong comparator arm, I believe that Opdivo plus Yervoy has the potential to become a standard of care for the first-line treatment of patients with unresectable or metastatic HCC.”

“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, in the release. “Today’s approval builds on the legacy of our dual immunotherapy and the value it has brought to patients for years. We are thrilled to add this indication for this important therapy—our second approval for Opdivo plus Yervoy in the gastrointestinal space this week alone—and look forward to providing a new first-line treatment option to patients in need.”

Adverse reactions associated with Opdivo plus Yervoy include pneumonitis, colitis, hepatitis and hepatotoxicity, endocrinopathies, nephritis with renal dysfunction, and dermatologic reactions. In addition, other adverse reactions may include infusion-related reactions; complications of allogeneic hematopoietic stem cell transplantation; embryo-fetal toxicity; and increased mortality in patients with multiple myeloma when Opdivo is added to a thalidomide analogue and dexamethasone.

Previously in 2020, FDA granted accelerated approval of the Opdivo (nivolumab) plus Yervoy (ipilimumab) combination based on results from a Phase I/II CheckMate-040 trial. It has been a second-line treatment for advanced HCC in patients who were previously treated with sorafenib. The new FDA approval expands the indication to a first-line treatment.

Reference

1. BMS. US Food and Drug Administration Approves Opdivo (nivolumab) plus Yervoy (ipilimumab) as a First-Line Treatment for Unresectable or Metastatic Hepatocellular Carcinoma. Press Release. April 11, 2025.