On June 20, 2022 Hepion Pharmaceuticals, Inc. (NASDAQ:HEPA), a clinical mid-stage biopharmaceutical company focused on Artificial Intelligence ("AI")-driven therapeutic drug development for the treatment of non-alcoholic steatohepatitis ("NASH") and hepatocellular carcinoma ("HCC"), reported that the U.S. Food and Drug Administration ("FDA") has granted Orphan Drug Designation to rencofilstat, a liver-targeting, orally administered, novel cyclophilin inhibitor, for the treatment of HCC (Press release, Hepion Pharmaceuticals, JUN 20, 2022, View Source [SID1234616098]).
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HCC is the most common form of liver cancer, accounting for 85-90% of all cases. NASH, viral hepatitis infection, and alcohol consumption all are major causes of HCC. Worldwide, it is estimated that over 800,000 people died from liver cancer in 2020, second only to lung cancer among all cancer-related deaths.1 The high mortality is largely due to the fact that only around half of all people who develop HCC (in developed countries) receive the diagnosis early enough to have an opportunity for therapeutic intervention. Additionally, recurrence rates are high, and effective treatment options remain limited.
"Orphan Drug Designation for rencofilstat in HCC represents a significant milestone for Hepion and its recognition by the FDA of the potential for rencofilstat to address a significant unmet medical need for patients suffering from this aggressive cancer," said Robert Foster, PharmD, PhD, Hepion’s CEO. "In addition to two Phase 2 studies in patients with NASH, we remain on track to initiate patient enrollment in a Phase 2a study of rencofilstat in HCC in the third quarter of 2022."
The FDA’s Orphan Drug Designation program provides orphan status to drugs or biologics intended for the prevention, diagnosis, or treatment of diseases that affect fewer than 200,000 people in the United States. Sponsors of medicines that are granted Orphan Drug Designation are entitled to certain incentives, including tax credits for qualified clinical trials, prescription drug user-fee exemptions, and potential seven-year marketing exclusivity upon FDA approval.