FDA approves Opdivo for previously treated hepatocellular carcinoma

The FDA approved nivolumab for the treatment of patients with hepatocellular carcinoma who underwent prior therapy with sorafenib.

“We are proud to bring the potential for clinically meaningful responses with immuno-oncology therapy to these advanced-stage hepatocellular carcinoma patients, who have had limited treatment options for years,” Chris Boerner, president of U.S. commercial business at Bristol-Myers Squibb, said in a company-issued press release. “[This] approval marks an important step toward our mission of delivering transformational medicines to treat conditions with a high unmet need.”

The FDA granted accelerated approval to nivolumab (Opdivo, Bristol-Myers Squibb) — an anti-PD-1 antibody — based on tumor response rate and response durability data from the CheckMate -040 trial.

The open-label, multicenter phase 1/phase 2 trial evaluated 3 mg/kg nivolumab IV every 2 weeks in 154 patients with hepatocellular carcinoma who previously received sorafenib (Nexavar, Bayer).

Twenty-two patients (14.3%; 95% CI, 9.2-20.8) responded to treatment, including three (1.9%) who achieved complete response and 19 (12.3%) who achieved partial response.

Responses ranged from 3.2 months to 38.2 months, with a median time to response of 2.8 months.

Overall, 91% of responding patients demonstrated responses of 6 months or longer, and 55% had responses of 12 months or longer.

Based on modified RECIST criteria, researchers reported an overall response rate of 18.2% (95% CI, 12.4-25.2), with 3.2% of patients achieving complete response and 14.9% achieving partial response.

The most common adverse reactions included fatigue (38%), musculoskeletal pain (36%), abdominal pain (34%), pruritus (27%), diarrhea (27%), rash (26%), cough (23%) and decreased appetite (22%).

The most common treatment-emergent grade 3 or grade 4 adverse events included aspartate transaminase increase (18%), alanine transaminase increase (11%) and elevated bilirubin (7%). At least 2% of patients experienced pyrexia, ascites, back pain, general physical health deterioration, abdominal pain or pneumonia.

“In recent years, there has been growing interest in leveraging immuno-oncology knowledge and discoveries to add to the treatment options available for patients with advanced-stage liver cancer,” Anthony B. El-Khoueiry, MD, associate professor of clinical medicine at Keck School of Medicine of USC and USC Norris Comprehensive Cancer Center, as well as lead investigator on the CheckMate -040 trial, said in the release. “The approval of Opdivo provides us with an encouraging approach and a new treatment option for appropriate patients with HCC following prior systemic therapy.”