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FDA Approves Nivolumab for Hepatocellular Carcinoma

Published: Friday, Sep 22, 2017

The FDA has granted an accelerated approval to nivolumab (Opdivo) for the treatment of patients with hepatocellular carcinoma (HCC) following prior sorafenib (Nexavar), regardless of PD-L1 status. 

In the sorafenib-naïve group across both the dose expansion and escalation cohorts (n = 80), the ORR by BICR was 20% by RECIST v1.1 and 24% by mRECIST. The v1.1 analysis noted 1 complete response (1%), 15 partial responses (19%), and 27 patients with stable disease (34%). Median duration of response was 17 months.

In the treatment expansion cohort, the median overall survival (OS) was 15.6 months (95% CI, 13.2-18.9). The 12-month OS rate was 60% and the 18-month rate was 44%. In the sorafenib-naïve group, the median OS was 28.6 months with nivolumab. The 12- and 18-month OS rates were 73% and 57%, respectively.

Responses tended to occur early; 56% of sorafenib-naïve patients and 64% of sorafenib-experienced patients who responded did so within 3 months of initiation. Responses were ongoing in 50% of sorafenib-naïve patients and 39% of sorafenib-experienced patients.

Investigators assessed safety across the escalation and expansion phases. Twenty-three sorafenib-naïve patients (29%) experienced grade 3/4 treatment-related adverse events (TRAEs) and 78% experienced any-grade TRAEs. The most common (³10%) any-grade TRAEs were pruritus (24%), fatigue (20%), rash (16%), and diarrhea (13%). The most common (≥5%) grade 3/4 TRAEs were AST increased (9%), lipase increased (8%), amylase increased (6%), and ALT increased (5%).

In the sorafenib-experienced group, 77% of patients experienced any-grade TRAEs and 18% had grade 3/4 TRAEs. The most common (≥10%) any-grade TRAEs were fatigue (22%), pruritus (20%), rash (18%), and diarrhea (14%). The most common grade 3/4 TRAEs were fatigue (4%), AST increased (4%), lipase increased (4%).

Investigators observed 1 dose-limiting toxicity, a grade 2 hepatic impairment in the dose-escalation phase. Maximum tolerated dose was not reached. One sorafenib-experienced patient died due to treatment-related pneumonitis.

“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,” Chris Boerner, president, US Commercial, Bristol-Myers Squibb, said in a statement. “Today’s approval marks an important step toward our mission of delivering transformational medicines to treat conditions with a high unmet need.”

Approval for nivolumab is contingent upon findings from a larger trial. A phase III randomized trial of nivolumab versus sorafenib has been launched in the frontline setting, with an enrollment goal of 726 patients. The estimated primary completion date is October 2018 (NCT02576509).
Crocenzi TS, El-Khoueiry AB, Yau T, et al. Nivolumab (nivo) in sorafenib (sor)-naive and -experienced pts with advanced hepatocellular carcinoma (HCC): CheckMate 040 study.J Clin Oncol. 2017;35 (suppl; abstr 4013).


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