FDA Approves Zenocutuzumab for NRG1 Fusion-Positive Cholangiocarcinoma Under Priority Voucher Program

The FDA approved zenocutuzumab-zbco (Bizengri) for adult patients with NRG1 fusion-positive advanced, unresectable, or metastatic cholangiocarcinoma who have progressed on prior systemic therapy. This is the first approved treatment for this rare bile duct cancer subtype. The approval was based on the single-arm Phase 1/2 eNRGy trial, where 19 evaluable patients achieved a 36.8% overall response rate. Duration of response ranged from 2.8 to 12.9 months. No patients discontinued due to adverse events. This was the seventh approval under the FDA's Commissioner's National Priority Voucher pilot program. Bizengri, developed by Partner Therapeutics (Merus NV), targets HER3 to block NRG1-driven HER2 heterodimerization. It was already approved for certain advanced lung and pancreatic cancers.