Taiwanese biopharma company, OBI Pharma, has announced that the US Food and Drug Administration (FDA) has granted orphan drug designation for its treatment of hepatocellular carcinoma (HCC), OBI-3424.
Liver cancer
“The orphan drug designation for OBI-3424 by the FDA is a significant step in the development of this drug candidate,” noted Amy Huang, general manager of OBI Pharma. “OBI-3424 is intended to treat a devastating form of liver cancer with limited therapeutic options. We are excited that the FDA has recognised the need to develop novel targeted therapeutic agents such as OBI-3424 in the fight against this disease.”
The drug candidate, OBI-3424, is a first-in-class DNA alkylating cancer therapeutic agent that targets aldo-keto reductase 1C3 (AKR1C3) overexpressing cancers. A Phase I/II clinical trial is already underway to assess the drug in patients with solid tumours — including HCC and castrate-resistant prostate cancer (CRPC).
HCC is a form of liver cancer associated with various stages of malignant growth in the liver. It has a low survival rate of around 12% for five years, making it the third major leading cause of cancer-related deaths worldwide. Additionally, patients with HCC have a high risk of developing treatment resistance to the standard of care therapies.