Bayer announced that the U.S. Food and Drug Administration (FDA) approved Stivarga (regorafenib) tablets for the second-line treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with Nexavar (sorafenib). Stivarga is the only treatment to demonstrate significant improvement in overall survival in second-line HCC patients. In the RESORCE trial, Stivarga was shown to provide a statistically significant and clinically meaningful improvement in overall survival (OS) versus placebo; the median OS was 10.6 [(n=379) (CI 9.1, 12.1)] vs 7.8 [(n=194) (CI 6.3, 8.8)] months, respectively (HR 0.63, 95% CI 0.50-0.79; p<0.0001). This translates to a 37% reduction in the risk of death. The number of deaths in each arm included 233 of 379 (62%) with Stivarga and 140 of 194 (72%) with placebo.
Stivarga is an oral inhibitor of multiple kinases involved in normal cellular functioning and in pathological processes such as oncogenesis, tumor angiogenesis, metastasis and tumor immunity. The FDA’s approval is based on data from the international, multicenter, placebo-controlled Phase III RESORCE [REgorafenib after SORafenib in patients with hepatoCEllular carcinoma] trial, which investigated patients with HCC whose disease had progressed during treatment with Nexavar. The most frequently observed adverse drug reactions (≥30%) in STIVARGA-treated patients vs placebo-treated patients in HCC, respectively, were: pain (55% vs 44%), HFSR/PPE (51% vs 7%), asthenia/fatigue (42% vs 33%), diarrhea (41% vs 15%), hypertension (31% vs 6%), infection (31% vs 18%), decreased appetite and food intake (31% vs 15%).
“Bayer is proud to have played a significant role in the treatment of hepatocellular carcinoma,” said Robert LaCaze, executive vice president and head of the Oncology Strategic Business Unit at Bayer. “We first embarked on our scientific research in this area 20 years ago and we have remained steadfast in our mission to deliver new treatment options to these patients. We could not have done it alone: we would like to thank the patients, caregivers and investigators for their participation and engagement in the study.”
Thomas F. Nealon III, national board chair and chief executive officer of the American Liver Foundation, commented, “Given that the incidence of liver cancer continues to rise, we applaud the efforts of Bayer and the RESORCE study investigators for ushering in this much-needed treatment option for patients with liver cancer.”
The approval of Stivarga in liver cancer marks the third time that this therapy has been granted FDA approval on a priority basis. The FDA granted Fast Track designation to Stivarga in this indication, which is an expedited program designed to facilitate development and review of drugs to address unmet medical need in the treatment of a serious or life-threatening condition. The FDA also granted Orphan Drug Designation (ODD) to Stivarga in HCC. The ODD program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases and disorders.
About Hepatocellular Carcinoma
Hepatocellular carcinoma (HCC) is the most common form of liver cancer and represents approximately three quarters of liver cancers in the United States. It is estimated that more than 40,000 people in the U.S. will be diagnosed with liver cancer in 2017. Liver cancer is still on the rise and death rates are increasing faster than any other type of cancer.3 In the United States, the incidence of liver cancer has more than tripled since 1980 and nearly 29,000 deaths from liver cancer are expected in 2017. In 2012, 782,000 men and women were diagnosed with liver cancer and approximately 746,000 people died of liver cancer worldwide.5 Globally, it is the second leading cause of cancer-related deaths.
About Stivarga
In April 2017, Stivarga was approved for use in patients with hepatocellular carcinoma who have been previously treated with Nexavar. In the United States, Stivarga is also indicated for the treatment of patients with metastatic colorectal cancer (CRC) who have been previously treated with fluoropyrimidine-, oxaliplatin- and irinotecan-based chemotherapy, an anti-VEGF therapy, and, if RAS wild-type, an anti-EGFR therapy. It is also indicated for the treatment of patients with locally advanced, unresectable or metastatic gastrointestinal stromal tumor (GIST) who have been previously treated with imatinib mesylate and sunitinib malate.
Regorafenib is a compound developed by Bayer. In 2011, Bayer entered into an agreement with Onyx, now an Amgen subsidiary, under which Onyx receives a royalty on all global net sales of regorafenib in oncology.