Eisai Co., Ltd. (Headquarters: Tokyo, CEO: Haruo Naito, “Eisai”) announced that it has submitted applications to the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for its in-house discovered and developed anticancer agent lenvatinib mesylate (lenvatinib) for the treatment of hepatocellular carcinoma (HCC). This follows the application in Japan. Lenvatinib for the treatment of HCC is designated as an orphan drug by the FDA.

This application is based on the results of the REFLECT study (Study 304), a multicenter, open-label, randomized, global Phase III trial comparing the efficacy and safety of lenvatinib versus sorafenib, a standard treatment for HCC, as a first-line treatment for 954 patients with unresectable HCC.

In the REFLECT study, lenvatinib met the primary endpoint and demonstrated an overall survival (OS) treatment effect by the statistical confirmation of non-inferiority compared to sorafenib. Developing first-line treatments for HCC is challenging, and over the past 10 years, four previous first-line Phase III studies investigating other agents compared to sorafenib have failed to achieve this endpoint in OS.

Additionally, lenvatinib showed highly statistically significant and clinically meaningful improvements in the secondary endpoints of Progression Free Survival (PFS), Time To Progression (TTP), and Objective Response Rate (ORR). In this study, the five most common adverse events observed in the lenvatinib arm were hypertension, diarrhea, decreased appetite, weight loss and fatigue, which is consistent with the known side-effect profile of lenvatinib.

Liver cancer is the second leading cause of cancer related death and is estimated to be responsible for 750,000 deaths per year globally (27,000 per year in the US, 62,000 per year in Europe), with 780,000 cases newly diagnosed each year (30,000 per year in the US, 63,000 per year in Europe). HCC accounts for 85% to 90% of liver cancer cases. Treatment options for unresectable HCC are limited and the prognosis is very poor, making this an area of high unmet medical need.

Lenvatinib is approved as a treatment for refractory thyroid cancer in over 50 countries, including the United States, Japan, and in Europe, under the brand name Lenvima?. Additionally, lenvatinib in combination with everolimus is approved for the treatment of renal cell carcinoma (RCC) in over 35 countries, including the United States and in Europe. In Europe, lenvatinib was launched under the brand name Kisplyx? for RCC.

Eisai positions oncology as a key therapeutic area, and is aiming to discover revolutionary new medicines with the potential to cure cancer. Eisai is committed to exploring the potential clinical benefits of lenvatinib as it seeks to contribute further to addressing the diverse needs of, and increasing the benefits provided to patients with cancer, their families, and healthcare providers.