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European NewsGenoscience Pharma received Orphan Drug Designation for hepatocellular carcinoma drugFrench clinical-stage biotech company Genoscience has received FDA Orphan Drug Designation (ODD) for its lead candidate ezurpimtrostat. ODD qualifies the drug for a period of seven years of market exclusivity after approval.Hepatocellular carcinoma (HCC) is a largely fatal cancer, with the median survival time for patients being between four and eight months. A combination treatment of atezolizumab and bevacizumab has more than doubled this timeline, however progression-free survival remains short.Ezurpimtrostat is a first-in-class, first-in-human autophagy inhibitor whose anticancer activity is linked to PPT-inhibition, both as a solo treatment and in combination with immune checkpoint inhibitors. When tested in HCC in vivo models, ezurpimtrostat showed high liver tropism and potent anti-tumour activity against a panel of human cancer cells.The ODD was granted after positive data from a phase 1 trial on primary and secondary liver tumours, where ezurpimtrostat was shown to be both feasible and well-tolerated as a monotherapy. A phase 2b ABE-Liver trial will now be conducted to test it in combination with an anti-PDL1 and an anti-angiogenic in up to 196 patients.“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. We have recently launched our phase 2b clinical trial using ezurpimtrostat in conjunction with the standard atezolizumab/bevacizumab treatment. We are looking forward to sharing the intermediate results in 2024,” Professor Philippe Halfon, CEO of Genoscience Pharma commented.