Tempest Announces It Has Received Orphan Drug Designation From European Medicines Agency For Its Amezalpat For Treatment Of Patients With Hepatocellular Carcinoma

Author: Benzinga Newsdesk | June 05, 2025 08:08am

• EMA Orphan Drug Designation (ODD) builds on U.S. Food & Drug Administration (FDA) ODD and Fast Track Designation, underscoring the urgent need for new treatment options
  
   
• The multiple regulatory designations were granted following strong positive results from a global randomized Phase 1b/2 study in first-line HCC demonstrating superior outcomes for amezalpat combination therapy across multiple study endpoints, including overall survival in both the entire population and key subpopulations, when compared to standard of care alone

- Tempest Therapeutics, Inc. (NASDAQ:TPST), a clinical-stage biotechnology company with a pipeline of first-in-class1 targeted and immune-mediated therapeutics to fight cancer, today announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) to amezalpat (TPST-1120), an oral, small molecule, selective PPAR⍺ antagonist for the treatment of patients with hepatocellular carcinoma (HCC).

"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," said Stephen Brady, president and chief executive officer of Tempest. "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."

The company announced earlier this year that the FDA had granted both ODD and Fast Track Designation (FTD) to amezalpat to treat patients with HCC. These three designations follow positive data across multiple key study efficacy and safety endpoints from a global, randomized Phase 1b/2 clinical study evaluating amezalpat plus standard-of-care atezolizumab and bevacizumab versus atezolizumab and bevacizumab alone in the first-line treatment of patients with unresectable or metastatic HCC. Notable positive outcomes of the randomized comparison include a six-month improvement in median overall survival (OS) with a hazard ratio (HR) of 0.65 for patients receiving the amezalpat combination therapy. In addition, a survival benefit from patients receiving amezalpat was preserved in key sub-populations including PD-L1 negative disease, which is consistent with amezalpat's proposed mechanism of action to target both the tumor cells directly and the patient's immune system.

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