FDA Grants Orphan Drug Status To Tempest’s Amezalpat For Liver Cancer Treatment

Tempest Therapeutics, Inc. is a biotechnology company that develops innovative Immune-mediated cancer therapies. The firm has made an announcement that the U.S. Food and Drug Administration (FDA) has granted an Orphan Drug Designation (ODD) status to amezalpat (TPST-1120). This regulatory milestone underscores the potential of amezalpat to address critical unmet needs in liver cancer treatment.

Amezalpat is an oral, selective PPAR⍺ antagonist designed to treat patients with hepatocellular carcinoma (HCC), a type of liver cancer. This designation comes on the heels of encouraging results from a global Phase 1b/2 clinical trial. The study evaluated the combination of amezalpat with standard-of-care treatments atezolizumab and bevacizumab against the standard treatments alone in patients with unresectable or metastatic HCC.

Sam Whiting, M.D., Ph.D., chief medical officer and head of R&D of Tempest, commented, “Receiving orphan drug designation for amezalpat to treat HCC underscores the critical need for new treatment options for patients suffering from this historically hard-to-treat disease. Tempest is dedicated to developing groundbreaking cancer treatments that will improve patients’ lives, and with broad agreement in hand from both the FDA and EMA, the team continues to prepare for a pivotal phase 3 study for amezalpat in first-line HCC patients.”

Patients receiving the amezalpat combination therapy demonstrated significant benefits, including a six-month improvement in median overall survival (OS) with a hazard ratio (HR) of 0.65 and a higher objective response rate (ORR) of 30% compared to 13% in the control group. Importantly, the survival benefits of amezalpat were consistent across key subgroups, including those with PD-L1 negative disease and b-catenin mutations. These results are evaluated based on the therapy's dual mechanism of action, which targets both tumour cells and the patient’s immune system.