Opdivo First Immunotherapy Approved for Hepatocellular Carcinoma

December 2017, Vol 7, No 12 - FDA News

On September 22, 2017, the FDA granted accelerated approval to nivolumab (Opdivo, Bristol-Myers Squibb) for the treatment of hepatocellular carcinoma (HCC)—the most common type of liver cancer—in patients who previously received sorafenib treatment. Opdivo received priority review for this indication.

“We are proud to bring the potential for clinically meaningful responses with Immuno-Oncology therapy to these advanced-stage HCC patients, who have had limited treatment options for years,” said Chris Boerner, the president of US commercial business at Bristol-Myers Squibb. “Today’s approval marks an important step toward our mission of delivering transformational medicines to treat conditions with a high unmet need.”

The approval of nivolumab for HCC was based on the data for a subgroup of 154 patients from the multicenter, open-label CheckMate-040 clinical trial of patients with HCC and Child-Pugh A cirrhosis, with or without active hepatitis B or C, whose diseases progressed with or who were intolerant of sorafenib.

The efficacy end points were overall response rate (ORR) and duration of response. Among the 22 responders, the ORR was 14.3% (95% confidence interval, 9.2%-20.8%) and the duration of response ranged from 3.2 months to >38.2 months; 91% of patients had responses that lasted ≥6 months, and 55% had responses lasting ≥1 years.

The most common (>20%) adverse reactions in this study included fatigue, rash, musculoskeletal pain, pruritus, diarrhea, nausea, asthenia, cough, dyspnea, constipation, decreased appetite, back pain, arthralgia, upper respiratory infection, and pyrexia; 49% of patients had serious adverse reactions. Grade 3 or 4 adverse events included AST (18%), ALT (11%), and bilirubin (7%).