Tempest’s Amezalpat Receives European Medicines Agency Orphan Drug Designation For Treating Hepatocellular Carcinoma

Tempest Therapeutics, Inc. announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) to its lead candidate, amezalpat (TPST-1120), for the treaatment of hepatocellular carcinoma (HCC), the most common type of liver cancer. Amezalpat is an oral, small-molecule antagonist of the PPAR⍺ pathway and is designed to target both tumor metabolism and immune evasion mechanisms. This European designation follows earlier U.S. regulatory milestones, where the FDA granted both Orphan Drug and Fast Track designations to amezalpat for the same indication. 

“We’re incredibly pleased to receive Orphan Drug Designation from the EMA, building on the momentum of regulatory support we've already received from the FDA. These designations reflect the significant unmet need in liver cancer and reinforce our belief in the potential of amezalpat to make a meaningful difference for patients and families affected by this devastating disease,” said Stephen Brady, president and chief executive officer of Tempest. 

These recognitions are based on promising results from a global Phase 1b/2 clinical trial comparing amezalpat in combination with standard therapies (atezolizumab and bevacizumab) against the standard treatment alone. The combination demonstrated a notable six-month improvement in median overall survival, with a hazard ratio of 0.65. Importantly, the survival benefit of amezalpat was consistent across key patient subgroups, including those with PD-L1 negative tumors, a population often resistant to immunotherapy.