FDA Approves Nivolumab, Ipilimumab for Patients with Hepatocellular Carcinoma

The FDA has approved nivolumab plus ipilimumab injections for intravenous use to treat hepatocellular carcinoma in patients who have been previously treated with sorafenib.

The FDA has approved nivolumab (Opdivo, Bristol-Myers Squibb) 1 mg/kg plus ipilimumab (Yervoy, Bristol-Myers Squibb) 3 mg/kg injections for intravenous use to treat hepatocellular carcinoma (HCC) in patients who have been previously treated with sorafenib.

The FDA granted this indication accelerated approval based on overall response rate and duration of response seen in the nivolumab plus ipilimumab cohort of the phase 1/2 CheckMate-040 trial. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

“HCC is an aggressive disease in need of different treatment approaches,” said lead investigator Anthony B. El-Khoueiry, MD, in a press release. “The overall response rate observed in the [treatment] cohort of the CheckMate-040 trial underscores the potential of this dual immunotherapy as a possible treatment option for patients.”

After a minimum follow up of 28 months in the CheckMate-040 cohort of HCC patients previously treated with sorafenib, 33% of patients responded to treatment with the combination therapy, and 8% had a complete response and 24% had a partial response. Duration of responses ranged from 4.6 to 30.5+ months, with 88% lasting at least 6 months, 56% at least 12 months, and 31% at least 24 months.

Nivolumab plus ipilimumab is the only dual immunotherapy approved by the FDA in this setting. The therapy features a potentially synergistic mechanism of action that targets 2 different checkpoints (PD-1 and CTLA-4) and works in complementary ways, according to the press release.

The combination therapy was granted Breakthrough Therapy Designation for this indication and Priority Review from the FDA.


  • U.S. Food and Drug Administration Approves Opdivo® (nivolumab) + Yervoy® (ipilimumab) for Patients with Hepatocellular Carcinoma (HCC) Previously Treated with Sorafenib [news release]. Bristol Myers Squibb website. Published March 11, 2020. https://news.bms.com/press-release/corporatefinancial-news/us-food-and-drug-administration-approves-opdivo-nivolumab-ye-0. Accessed March 12, 2020.

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