FDA Grants Orphan Drug Designation to Ezurpimtrostat for HCC Treatment

The FDA has granted an orphan drug designation (ODD) to ezurpimtrostat (GNS561), a PPT-1 (palmitoyl protein thioesterase-1) inhibitor for the treatment of patients with hepatocellular carcinoma (HCC), according to an announcement from Genoscience Pharma.

Philippe Halfon, MD, PhD, chief executive officer of Genoscience Pharma, stated, "FDA Orphan Drug Designation is a significant milestone for both Genoscience and for our product, ezurpimtrostat. It recognizes that our treatment has the potential to improve the lives of individuals living with HCC." The company has recently launched a phase 2b clinical trial using ezurpimtrostat in conjunction with the standard atezolizumab/bevacizumab treatment, with intermediate results expected in 2024.

Liver cancer is the sixth most common cancer worldwide, with significant mortality rates. The FDA's ODD supports the development of treatments for rare diseases affecting fewer than 200,000 people in the United States. Without treatment, the median survival for patients with advanced progressive HCC ranges from 4 to 8 months. The combination of atezolizumab and bevacizumab has improved life expectancy, but new treatments are needed to extend progression-free survival.

Ezurpimtrostat is a first-in-class autophagy inhibitor with anticancer activity through PPT-1 inhibition. It has shown high liver tropism and potent anti-tumor activity in preclinical models and human cancer cell lines. Preliminary data from a phase 1b trial demonstrated that ezurpimtrostat is feasible and well-tolerated, with manageable adverse effects. The phase 2b ABE-Liver trial is currently enrolling patients to evaluate ezurpimtrostat in combination with anti-PD-L1 and anti-angiogenic agents as a first-line treatment for HCC.