FDA Grants Accelerated Approval to Opdivo-Yervoy Combo for Advanced HCC

The Food and Drug Administration (FDA) granted accelerated approval to the combination use of Opdivo (nivolumab) and Yervoy (ipilimumab) for the treatment of patients with advanced hepatocellular carcinoma (HCC) who were previously treated with Nexavar (sorafenib).

This approval, which is the only dual immunotherapy approved by the FDA in this setting, was based on the overall response rate and duration of response in this combination from results in the phase 1/2 CheckMate-040 trial. This, according to Bristol Myers Squibb.

“HCC is an aggressive disease in need of different treatment approaches,” said Dr. Anthony B. El-Khoueiry, lead investigator and associate professor of clinical medicine and phase I program director at the Keck School of Medicine, University of Southern California (USC) and the USC Norris Comprehensive Cancer Center, in a press release. “The overall response rate observed in the Opdivo-Yervoy cohort of the CheckMate-040 trial underscores the potential of this dual immunotherapy as a possible treatment option for patients.”

In the trial, researchers evaluated the Opdivo-Yervoy combination in 49 patients with advanced HCC who progressed on or were intolerant to previous treatment with Nexavar. Data from the phase 1/2 open label, multi-cohort CheckMate-040 trial was presented at the 2019 American Society for Clinical Oncology (ASCO) Annual Meeting.

The researchers found that 33% of the patients on the trial responded to the Opdivo-Yervoy combination treatment, with 8% showing a complete response and 24% exhibiting a partial response. The duration of responses lasted from 4.6 to greater than 30.5 months and 88% of patients on the trial saw a duration of response that lasted at least six months.

Serious side effects were seen in 59% of the patients, with 29% discontinuing their treatment and 65% delaying it due to side effects. Serious side effects included pyrexia, diarrhea, anemia, increased aspartate aminotransferase, adrenal insufficiency, ascites, esophageal varices hemorrhage, hyponatremia, increased blood bilirubin and pneumonitis. The most common of these side effects was rash, pruritus, musculoskeletal pain, diarrhea, cough, decreased appetite, fatigue, pyrexia, abdominal pain, headache, nausea, dizziness, hypothyroidism and weight decrease.

Check back for continued coverage on what you need to know about this approval.