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FDA Accepts Biologics License Application For Nivolumab Plus Ipilimumab to Treat Hepatocellular Carcinoma
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The FDA assigned the combination a Prescription Drug User Fee Act goal date of April 21, 2025.
The FDA has accepted the supplemental biologics license application (sBLA) for nivolumab (Opdivo; Bristol Myers Squibb), a programmed death-1 (PD-1) immune checkpoint inhibitor, in combination with the human monoclonal antibody ipilimumab (Yervoy; Bristol-Myers Squibb) in adult patients with unresectable hepatocellular carcinoma (HCC), according to a news release.1
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Acceptance of the drug combination was informed by results from the phase 3 CheckMate -9DW trial, in which the combination demonstrated statistically significant and clinically meaningful improvement of overall survival (OS) compared to a choice of lenvatinib or sorafenib.1
“With the number of individuals diagnosed with HCC in the United States increasing over the last decade, new treatment options are urgently needed,” Dana Walker, vice president and global program lead at Bristol Myers Squibb, said in the news release. “We look forward to working with the FDA to advance our application to potentially bring a new first-line treatment option to patients.”1
CheckMate -9DW was randomized and open-label and enrolled 668 patients to either receive infusions of nivolumab plus ipilimumab for up to 4 doses, followed by monotherapy with nivolumab, oral capsules of single-agent lenvatinib, or sorafenib for control patients. The primary end point is OS, with notable secondary end points including objective response rate and the time to deterioration of symtpoms.1
This drug combination has been previously established as a second-line treatment for HCC and is being investigated in a series of CheckMate trials for multiple other types of cancers. In one trial, CheckMate-8HW, results indicated that the nivolumab-ipilimumab combination demonstrated significant improvements in progression-free survival in patients with metastatic colorectal cancer.2
In another trial, CheckMate 227, the combination met both primary end points, demonstrating durable, long-term survival benefits compared to chemotherapy as a first-line treatment for patients with metastatic non-small cell lung cancer. The combination is now on track to being a first-line treatment for a cancer that is the most common form of primary liver cancer.1,3
Often diagnosed in patients at an older age, HCC accounts for 75% to 85% of all liver cancers. Given the age of many patients, effective treatment options are typically limited and are associated with poor health outcomes. In addition, up to 70% of patients will experience a recurrence of HCC within 5 years of diagnosis.1
Causes of HCC vary. Most cases are caused by hepatitis B virus or hepatitis C virus infections, but cases due to metabolic syndrome and nonalcoholic steatohepatitis (NASH) are becoming more common and are expected to contribute to higher rates of HCC.1
Given NASH—and liver disease in general—can disproportionately affect lower income patients and minority groups, it is essential that any new drugs being developed and manufactured are utilized to reduce health care gaps between populations.4
Ipilimumab received FDA approval in March 2011 for patients with unresectable or metastatic melanoma, further emphasizing the cross-disease potential for the drug. Furthermore, nivolumab was the first PD-1 immune checkpoint inhibitor to receive regulatory approval anywhere globally.1
In lieu of the sBLA acceptance, the FDA assigned the combination a Prescription Drug User Fee Act (PDUFA) goal date of April 21, 2025.1
