On May 10, 2019, the Food and Drug Administration approved ramucirumab (CYRAMZA®, Eli Lilly and Company) as a single agent for hepatocellular carcinoma (HCC) in patients who have an alpha fetoprotein (AFP) of ≥ 400 ng/mL and have been previously treated with sorafenib.
Approval was based on REACH 2 (NCT02435433), a multinational, randomized, double-blind, placebo-controlled, multicenter study in 292 patients with advanced HCC with AFP ≥ 400 ng/mL who had disease progression on or after sorafenib or who were intolerant. Patients were randomized (2:1) to receive ramucirumab 8 mg/kg plus best supportive care (BSC) or placebo plus BSC every 2 weeks as an intravenous infusion until disease progression or unacceptable toxicity.
The trial’s primary endpoint was overall survival (OS). The estimated median OS was 8.5 months (7.0, 10.6) for patients receiving ramucirumab and 7.3 months (5.4, 9.1) for those receiving placebo (HR 0.71; 95% CI: 0.53, 0.95; p=0.020).
The most common adverse reactions observed in patients with HCC receiving single-agent ramucirumab (≥ 15% and ≥ 2% higher incidence than placebo) were fatigue, peripheral edema, hypertension, abdominal pain, decreased appetite, proteinuria, nausea, and ascites. The most common laboratory abnormalities (≥ 30% and a ≥ 2% higher incidence than placebo) were hypoalbuminemia, hyponatremia, and thrombocytopenia.
The recommended ramucirumab dose is 8 mg/kg administered intravenously every 2 weeks.
Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 1-800-FDA-1088.
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