FDA Approves Opdivo Plus Yervoy for First-Line Treatment of Unresectable or Metastatic Hepatocellular Carcinoma

The U.S. Food and Drug Administration (FDA) has approved Bristol Myers Squibb's Opdivo (nivolumab) in combination with Yervoy (ipilimumab) for the first-line treatment of adult patients with unresectable or metastatic hepatocellular carcinoma (HCC). This approval marks a significant advancement in the treatment landscape for liver cancer patients, offering a new immunotherapy option in the first-line setting.

Breakthrough in HCC Treatment

The approval is based on results from the Phase 3 CheckMate-9DW trial, which demonstrated superior overall survival (OS) and overall response rate (ORR) for Opdivo plus Yervoy compared to the current standard of care. In the trial, patients receiving the combination therapy showed a median OS of 23.7 months versus 20.6 months for those treated with lenvatinib or sorafenib, reducing the risk of death by 21%.

Dr. Aiwu Ruth He, a CheckMate-9DW study investigator, commented on the significance of this approval: "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."

Efficacy and Safety Profile

The combination therapy showed impressive efficacy results:

• 38% of patients treated with Opdivo plus Yervoy were still alive at 3 years, compared to 24% in the comparator arm
• ORR of 36.1% for Opdivo plus Yervoy versus 13.2% for lenvatinib or sorafenib
• Median duration of response of 30.4 months for the combination therapy compared to 12.9 months for the comparator arm

The safety profile of Opdivo plus Yervoy is well-established, with no new safety signals identified in the CheckMate-9DW trial. However, the treatment is associated with immune-mediated adverse reactions, which can be severe or fatal in some cases.

Implications for HCC Patients

Hepatocellular carcinoma is the most common form of liver cancer in adults and is often diagnosed at an advanced stage. With limited effective treatment options available, this approval provides a new first-line option for patients facing this challenging diagnosis.

Wendy Short Bartie, senior vice president of Oncology Commercialization at Bristol Myers Squibb, stated, "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 and look forward to providing a new first-line treatment option to patients in need."

As the incidence of liver cancer continues to rise, with metabolic syndrome and metabolic dysfunction-associated steatohepatitis expected to contribute to increased rates of HCC, this approval represents a significant step forward in addressing an urgent medical need.