FDA Approves Nivolumab Plus Ipilimumab for Patients With Unresectable or Metastatic Hepatocellular Carcinoma
On April 11, 2025, the US Food and Drug Administration approved nivolumab plus ipilimumab for first-line treatment of adult patients with unresectable or metastatic hepatocellular carcinoma. This approval was based on results from the CheckMate-9DW trial.
In this open-label trial, 668 patients with unresectable or metastatic hepatocellular carcinoma who had not received any prior systemic therapy were randomized on a 1-to-1 basis to receive either 1 mg/kg nivolumab with 3 mg/kg ipilimumab every 3 weeks for a maximum of 4 doses, followed by 480 mg single-agent nivolumab every 4 weeks, or investigator’s choice of either lenvatinib or sorafenib. The primary efficacy measure was overall survival (OS) with overall response rate (ORR) by blinded independent central review as an additional measure.
The median OS was 23.7 months in the nivolumab-ipilimumab arm vs 20.6 months in the lenvatinib or sorafenib arm (hazard ratio, 0.79; P < .0180). The ORR was 36.1% in the nivolumab-ipilimumab arm vs 13.2% in the lenvatinib or sorafenib arm (P < .0001). The most common adverse events occurring in > 20% of patients were rash, pruritus, fatigue, and diarrhea.
The recommended dose for nivolumab is 1 mg/kg with ipilimumab 3 mg/kg given intravenously every 3 weeks for a maximum of 4 doses, followed by single-agent nivolumab at 240 mg intravenous every 2 weeks or at 480 mg intravenously every 4 weeks.
Source:
FDA approves nivolumab with ipilimumab for unresectable or metastatic hepatocellular carcinoma. Accessed April 10, 2025. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-nivolumab-ipilimumab-unresectable-or-metastatic-hepatocellular-carcinoma
