Elevar Therapeutics Receives EMA Orphan Drug Status for First-Line Hepatocellular Carcinoma Therapy

On August 1, 2024, Elevar Therapeutics, Inc., a subsidiary of HLB Co., Ltd., announced that the European Medicines Agency (EMA) has granted Orphan Medicinal Product Designation to rivoceranib in combination with camrelizumab for the first-line treatment of Unresectable Hepatocellular Carcinoma (uHCC).

Chris Galloway, M.D., Senior Vice President of Clinical and Medical Affairs at Elevar Therapeutics, expressed that the EMA's designation highlights the urgent need for new liver cancer treatments. The orphan status aids Elevar in its mission to provide a novel first-line systemic treatment option for hepatocellular carcinoma (HCC) patients in the European Union (EU), an area where the disease is a significant cause of cancer-related deaths.

The EMA grants orphan designation based on several criteria: the medicine must target a life-threatening or chronically debilitating disease; the prevalence of the condition in the EU must be less than 5 in 10,000, or it must be unlikely that the medicine's market revenue would justify the investment needed for its development; and there should be no satisfactory existing method of diagnosis, prevention, or treatment, or the new medicine must offer significant benefits over existing methods.

Medicines with orphan designation in the EU enjoy several incentives, including access to centralized authorization that results in a single opinion and decision from the European Commission valid across all EU Member States. Additionally, once approved, these medicines benefit from ten years of market protection from competition with similar products for the same indication.

Every year, over 800,000 people globally are diagnosed with liver cancer, leading to more than 700,000 deaths annually. HCC, the most common type of primary liver cancer, often arises in individuals with chronic liver inflammation due to viral or non-viral causes. HCC has a poor prognosis and limited treatment options, highlighting the ongoing need for new therapies.

Rivoceranib, a tyrosine kinase inhibitor (TKI), is a potent inhibitor of the vascular endothelial growth factor receptor (VEGFR), a primary pathway for tumor angiogenesis. Inhibiting VEGFR is a clinically validated approach to reducing tumor growth and disease progression. Rivoceranib is currently being studied both as a monotherapy and in combination with other treatments like chemotherapy and immunotherapy for various solid tumors. Clinical trials include studies on uHCC (in combination with camrelizumab), gastric cancer, adenoid cystic carcinoma, and colorectal cancer.

Rivoceranib was first approved in China in 2014 for gastric cancer and later in 2023 for uHCC in combination with camrelizumab. It has been studied in over 6,000 patients worldwide and has demonstrated a tolerable safety profile similar to other TKIs and VEGF inhibitors. Orphan drug designations have also been granted for rivoceranib in various countries, including the U.S., EU, and South Korea, for gastric cancer, adenoid cystic carcinoma, and uHCC. Elevar Therapeutics holds the global rights to rivoceranib outside of China and has partnered with HLB-LS for its development and marketing in South Korea.

Camrelizumab, developed by Hengrui Pharma, is a humanized monoclonal antibody targeting the PD-1 receptor. Blocking the PD-1/PD-L1 pathway has shown success in treating a variety of cancers. Camrelizumab has been studied in over 5,000 patients across 50 clinical trials for different tumor types, including liver, lung, gastric, and breast cancers. It is approved in China for several indications, including monotherapy for HCC and in combination with rivoceranib for uHCC.

In October 2023, Elevar licensed camrelizumab for worldwide commercialization, excluding Greater China and Korea, from Hengrui Pharma. Elevar Therapeutics, based in New Jersey with offices in South Korea, focuses on developing innovative treatments for complex and under-treated health conditions, particularly in oncology. Rivoceranib is their lead proprietary drug candidate.

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