Genfit (NASDAQ:GNFT) – GENFIT Obtains Orphan Drug Status from the FDA for its Liver Disease Candidate

The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to GENFIT GNFT drug candidate GNS5611 (ezurpimtrostat) for the treatment of cholangiocarcinoma.

Preclinical studies evaluating GNS561 have been completed and a phase 1b trial confirms 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 tumor cell growth in advanced cancers.

Dr. Mark Yarchoan, Associate Professor of Oncology at Johns Hopkins Medicine, said: “Cholangiocarcinoma is a rare cancer with a high mortality rate. Patients have limited treatment options, especially after first-line treatment. This is why new therapies are urgently needed and is one of the reasons GNS561 has been granted orphan drug designation by the FDA. There is a real way forward for new second-line treatment options for cholangiocarcinoma, and GNS561 represents a strong second-line therapeutic candidate and hope for patients.

The company is expected to begin a Phase 2 in Q4 2022 with subject dosing expected in Q1 2023.

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

The FDA is granting orphan designation to promote the development of a drug that is expected to have significant therapeutic advantage over existing treatments targeting a disease affecting 200,000 or fewer US patients each year.

Price Action: GENFIT shares are trading up around 11% at $4.90 on Tuesday during the after-market session.

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