Genoscience Pharma receives FDA Orphan Drug Designation for ezurpimtrostat to treat HepatoCellular Carcinoma (HCC)
Ezurpimtrostat could meet important unmet need in HCC, bringing new treatment options to patients worldwide
FDA designation significant milestone following launch of phase 2b ABE-Liver trial
Marseille, France, February 1, 2023 – Genoscience Pharma, a clinical-stage biotech company developing unique lysosomotropic drug candidates for the treatment of cancer, fibrosis and auto-immune diseases through autophagy modulation, today announces that its lead candidate, ezurpimtrostat, a PPT-1 (Palmitoyl Protein Thioesterase-1) inhibitor, has been granted Orphan Drug Designation (ODD) by the US Food and Drug Administration (FDA) for the treatment of HepatoCellular Carcinoma (HCC). This is an important milestone in the development of ezurpimtrostat, as well as for patients. ODD qualifies ezurpimtrostat for a potential seven years of market exclusivity after approval.
