Nivolumab

Bristol Myers Squibb (BMS) has announced that its dual immunotherapy combination has been approved by the US Food and Drug Administration (FDA) to treat patients with hepatocellular carcinoma (HCC), the most common type of liver cancer. Opdivo (nivolumab) plus Yervoy (ipilimumab) is now authorised by the regulator as a first-line treatment for adults with unresectable or advanced cases of the disease. Approximately 42,240 people in the US are expected to be diagnosed with liver cancer this year. HCC accounts for approximately 90% of all liver cancer cases and is often diagnosed at an advanced stage, at which point effective treatment options are limited. Opdivo plus Yervoy was previously granted accelerated approval by the FDA to treat advanced HCC in patients previously treated with sorafenib. The regulator’s latest decision converts this to full approval while expanding the indication into the first-line setting. The new authorisation was based on positive results from the phase 3 CheckMate-9DW trial, in which Opdivo plus Yervoy demonstrated a statistically significant and clinically meaningful improvement in overall survival and overall response rate compared to investigator’s choice of lenvatinib or sorafenib. Median overall survival was 23.7 months for Opdivo plus Yervoy compared to 20.6 months with lenvatinib or sorafenib, and objective response rate was 36.1% for BMS’ combination versus 13.2% for the comparator arm. Longer responses were also seen with Opdivo plus Yervoy, and no new safety signals were observed. CheckMate-9DW study investigator, Aiwu Ruth He, MedStar Georgetown University Hospital, said: “The availability of a new first-line treatment option that demonstrated a deep response can offer adults with this form of liver cancer long-term overall survival and may help address an unmet need. “Given the strength of evidence from the trial, especially considering the selection and performance of a strong comparator arm, I believe that Opdivo plus Yervoy has the potential to become a standard of care for the first-line treatment of patients with unresectable or metastatic HCC.” The announcement comes just days after the Opdivo/Yervoy combination was approved by the FDA to treat adult and paediatric patients aged 12 years and older with unresectable or metastatic microsatellite instability-high or mismatch repair deficient colorectal cancer.

The trial compared the combo against the investigator’s choice of tyrosine kinase inhibitor monotherapy in the unresectable or advanced hepatocellular carcinoma patient group. Credit: PanuShot/Shutterstock. The US Food and Drug Administration (FDA) has granted approval for Bristol Myers Squibb’s (BMS) Opdivo (nivolumab) in combination with Yervoy (ipilimumab) as a first-line treatment for adults with unresectable or metastatic hepatocellular carcinoma (HCC), a primary liver cancer. The approval is anchored in the outcomes from the open-label randomised Phase III CheckMate-9DW trial, in which the combination showed significant overall response rate (ORR) and overall survival (OS) against the comparator arm. The trial compared the combo against the investigator’s choice of tyrosine kinase inhibitor monotherapy – either lenvatinib or sorafenib in the unresectable or advanced HCC patient group, who had not undergone previous systemic therapy. Its primary endpoint is OS, with secondary endpoints objective response rate and time to symptom deterioration. The latest decision by the agency grants full approval to the regimen and extends its use to the first-line treatment setting, reflecting the CheckMate-9DW trial’s findings. BMS noted that the trial was not structured to independently evaluate Opdivo plus Yervoy against lenvatinib or sorafenib alone. BMS oncology commercialisation senior vice-president Wendy Short Bartie stated: “Bringing Opdivo plus Yervoy to patients with HCC in the first-line setting is a testament to our ongoing commitment to research and delivering important progress for people living with cancer. “Today’s approval builds on the legacy of our dual immunotherapy and the value it has brought to patients for years. We are thrilled to add this indication for this important therapy – our second approval for Opdivo plus Yervoy in the gastrointestinal space this week alone – and look forward to providing a new first-line treatment option to patients in need. ” In 2020, the combination received accelerated approval from the US regulator based on the Phase I/II CheckMate-040 trial outcomes. It had been recognised as a second-line treatment for advanced HCC patients treated with sorafenib. In March 2025, BMS agreed to acquire 2seventy bio, the company’s longtime cell therapy partner, in a $286m deal.