FDA Grants Orphan Drug Designation to Ezurpimtrostat (GNS561) for Hepatocellular Carcinoma (HCC) Treatment

Genoscience Pharma today announces that its lead candidate, ezurpimtrostat (GNS561), a PPT-1 (Palmitoyl Protein Thioesterase-1) inhibitor, has been granted Orphan Drug Designation by the U.S. FDA for the treatment of Hepatocellular Carcinoma (HCC). This is a significant milestone in the development of ezurpimtrostat for patients. ODD qualifies ezurpimtrostat for a potential seven years of market exclusivity after approval.

Publish Date: 08-02-2023 Source: Genoscience Pharma

Hepatocellular Carcinoma (HCC) is the most common type of primary liver cancer associated with liver cirrhosis, and it develops after long periods of chronic liver disease. Cirrhosis is characterised by a decrease in hepatocyte proliferation, which indicates that the liver’s regenerative capacity is exhausting. It causes an increase in fibrous tissue and the destruction of liver cells, which may eventually lead to the formation of cancerous nodules. Approximately 70%–90% of patients with HCC have an established background of chronic liver disease and cirrhosis, including chronic infection with hepatitis B virus (HBV), hepatitis C virus (HCV), alcoholic liver disease, and nonalcoholic steatohepatitis (NASH). Half of all HCC cases are caused by hepatitis B virus infection, with 25% caused by hepatitis C virus infection. Unresectable HCC is clinically a heterogeneous group of patients, including those with the intermediate stage (BCLC B) and advanced stage (BCLC C).

However, the current Hepatocellular Carcinoma (HCC) treatment market share, market uptake, and attribute analysis concerning the most potential emerging therapies (Namodenoson, AK104, ATG-008, etc..) has been provided under the market outlook section of the study covering 8 MM countries; The United States, EU5 (Germany, Spain, France, Italy, UK) Japan and China.

In terms of pharmacologic therapies, several pharmaceutical products are being approved and under different phases of development for the Hepatocellular Carcinoma treatment. The key companies in the advanced development stage are Can-Fite BioPharma, Akeso, Antengene Therapeutics Limited, etc.. targeting HCC.

Based on solid domain and business knowledge, Thelansis Knowledge Partners has published the market outlook forecast report on Hepatocellular Carcinoma (HCC) to provide a clear understanding of disease area background, epidemiology, current and future competitions, the country-specific standard of care, and the complete market forecast for 2022 to 2032.

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