FDA Approves BMS Immunotherapy Combo for Liver Cancer

Bristol Myers Squibb (BMS) has achieved a significant milestone with the recent approval by the US Food and Drug Administration (FDA) of its dual immunotherapy regimen for hepatocellular carcinoma (HCC), the most prevalent form of liver cancer. This regimen, consisting of Opdivo (nivolumab) and Yervoy (ipilimumab), is now sanctioned as a first-line therapy for adults with advanced or unresectable HCC.

Liver cancer is anticipated to affect approximately 42,240 individuals in the United States this year. Notably, HCC comprises about 90% of all liver cancer cases and is frequently identified at later stages, where treatment options become constrained. The new FDA approval represents a crucial advancement in addressing these challenges, offering a new avenue for initial treatment.

Previously, Opdivo combined with Yervoy was granted accelerated approval for treating advanced HCC in patients who had received prior treatment with sorafenib. The recent approval by the FDA not only extends this indication to include first-line treatment but also confirms the combination's full approval, marking a significant progression in therapeutic options for liver cancer patients.

The basis for this new approval is rooted in the positive outcomes of the phase 3 CheckMate-9DW trial. In this study, the combination of Opdivo and Yervoy exhibited a statistically and clinically meaningful enhancement in both overall survival and response rates, compared to the investigator’s choice of lenvatinib or sorafenib. Specifically, the median overall survival for patients treated with the Opdivo and Yervoy combination was 23.7 months, compared to 20.6 months for those on lenvatinib or sorafenib. Furthermore, the objective response rate for the BMS combination reached 36.1%, significantly higher than the 13.2% observed in the comparator group. Additionally, patients receiving the dual therapy experienced longer-lasting responses, without the emergence of new safety concerns.

Aiwu Ruth He, a CheckMate-9DW study investigator from MedStar Georgetown University Hospital, emphasized the importance of this new treatment option. She noted the deep responses achieved with the dual immunotherapy, which could potentially provide long-term survival benefits and address an unmet need for individuals with this type of liver cancer. Given the compelling evidence from the trial, particularly the strong performance of the comparator arm, Dr. He believes that Opdivo paired with Yervoy could establish itself as a standard of care for first-line treatment in patients with unresectable or metastatic HCC.

This approval comes shortly after the same combination received FDA approval for treating adult and pediatric patients aged 12 and older with unresectable or metastatic microsatellite instability-high or mismatch repair deficient colorectal cancer. This sequential approval underscores the expanding role and potential of Opdivo and Yervoy in cancer treatment, offering renewed hope for patients and healthcare providers in managing these challenging conditions.