GENFIT Gets FDA Orphan Drug Status For Liver Disease Candidate

 The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to GENFIT’s 

GNFT-1.78%+ Free Alerts

 drug candidate GNS5611 (ezurpimtrostat) for the treatment of cholangiocarcinoma.

The pre-clinical studies evaluating GNS561 has been completed and a Phase 1b trial confirming the rationale for targeting cholangiocarcinoma.

GNS561 (ezurpimtrostat) is a Palmitoyl Protein Thioesterase-1 (PPT-1)  inhibitor that blocks autophagy. Autophagy is activated in tumor cells in response to certain conditions, due to a tumor cell growth in advanced cancers.

Dr Mark Yarchoan, Associate Professor of Oncology at John Hopkins Medicine, commented: “Cholangiocarcinoma is a rare cancer with a high mortality rate. Patients have limited treatment options, particularly following first line therapy. This is why new therapies are urgently needed and is one of the reasons that GNS561 was granted Orphan Drug Designation by the FDA. There is a real path forward for new options for second line treatment in cholangiocarcinoma, and GNS561 represents a strong second-line therapy candidate and hope to patients.”

The company is expected to commence a Phase 2 in the fourth quarter 2022, and dosing of subjects is expected in the first quarter 2023.

Cholangiocarcinoma is a rare liver malignancy with high mortality and limited treatment options. It occurs mostly in people over the age of 50.

https://www.benzinga.com/general/biotech/22/09/28857097/genfit-gets-fda-orphan-drug-status-for-liver-disease-candidate