Taiwanese biopharma gains orphan drug designation from FDA

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.

“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.