Approval for the combination treatment was based on results from the Phase I/II CheckMate-040 trial in which Opdivo and Yervoy showed an overall response rate of 33% in this patient population.
The U.S. Food and Drug Administration (FDA) gave an accelerated greenlight to Bristol Myers Squibb’s combination of Opdivo and Yervoy as a treatment for hepatocellular carcinoma in patients who have been previously treated with Bayer’s Nexavar (sorafenib).
Approval for the combination treatment was based on results from the Phase I/II CheckMate-040 trial in which Opdivo and Yervoy showed an overall response rate of 33% in this patient population. Of those, 8% had a complete response and 24% showed a partial response. Duration of responses (DOR) ranged from 4.6 to 30.5 months, with 88% lasting at least six months, 56% at least 12 months and 31% at least 24 months, BMS said.
Anthony B. El-Khoueiry, BMS’ lead investigator and phase I program director at the Keck School of Medicine, University of Southern California, said the overall response rate observed in the Opdivo and Yervoy combination of the CheckMate-040 trial underscores the potential of this dual immunotherapy as a possible treatment option for patients.
Opdivo (nivolumab) and Yervoy (ipilimumab) are cornerstones of BMS immunology treatments. Opdivo, a programmed death-1 (PD-1) immune checkpoint inhibitor, has been approved for the treatment of multiple cancers, including patients with advanced renal cell carcinoma who have received prior anti-angiogenic therapy. Yervoy targets CTLA-4, a protein receptor that regulates the immune system.
The FDA gave the nod under accelerated approval. Continued approval in this indication may be contingent upon verification and description of clinical benefit in confirmatory trials, the company noted. The Opdivo and Yervoy combination is the first and only dual immunotherapy approved in this setting, BMS said. The company added that this is the fourth regulatory approval for cancer from the FDA for the combination treatment. The combination treatment had been previously granted both Breakthrough Therapy Designation for this indication and a Priority Review from the FDA.
Hepatocellular carcinoma (HCC) is the most common type of liver cancer in adults. HCC represents about 80% of all primary liver cancers. The disease occurs most often in people with chronic liver diseases, such as cirrhosis caused by hepatitis B or hepatitis C infection.
Adam Lenkowsky, general manager and head of U.S. Oncology, Immunology and Cardiovascular at Bristol Myers Squibb, said there is a critical need to provide patients with aggressive forms of cancer such as HCC with new treatment options that may “offer clinically meaningful and ultimately durable responses.” The approval, Lenkowsky said, builds on the company’s legacy in pioneering immunotherapy treatments.
“The incidence of liver cancer is rising in the United States, and HCC is the most common and aggressive form of the disease, “Andrea Wilson, president and founder of Blue Faery: The Adrienne Wilson Liver Cancer Association, said in a statement. “Today’s approval provides a new option for patients with HCC previously treated with sorafenib, giving the community more hope.”
Last year, Eli Lilly snagged regulatory approval for Cyramza as a single agent for the second-line treatment of patients with hepatocellular carcinoma (HCC) who have an alpha-fetoprotein (AFP) and have previously been treated with sorafenib. Also in 2019, Alameda, Calif.-based Exelixis, Inc. won approval for its HCC treatment, Cabometyx (cabozantinib).
