OBI Pharma announced on the 16th that it has received notification from the U.S. Food and Drug Administration (FDA) that its TROP2-targeted antibody–drug conjugate (ADC) candidate OBI-902 has been granted orphan drug designation for the treatment of cholangiocarcinoma, marking a major milestone in the company’s in-house ADC development strategy.

OBI-902 is OBI Pharma’s first self-developed novel ADC employing site-specific conjugation technology, and is among the few ADC new drugs in Taiwan to be advanced by a local team from mechanism design through to clinical development.

OBI Pharma initiated Phase I/II clinical trials of OBI-902 concurrently in the United States and Taiwan in August 2025, targeting patients with advanced solid tumors. The study aims to evaluate safety, pharmacokinetic (PK) profiles, and preliminary efficacy.

Cholangiocarcinoma is a rare but highly lethal malignancy, with fewer than 50,000 patients in the United States. Depending on disease stage, histological subtype, and tumor location, the five-year survival rate ranges from approximately 2% to 23%. To date, the U.S. FDA has not approved any ADC therapies for cholangiocarcinoma, and available treatment options remain limited, underscoring the urgent need for the development of new therapies.

Resource: 浩鼎新藥獲美孤兒藥認定