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FDA Approves First Drug for Rare Bile Duct Cancer
11 May
Summary
- Bizengri is the first drug approved for adults with advanced cholangiocarcinoma.
- The approval targets cancers with NRG1 gene fusion, a rare genetic anomaly.
- This marks the seventh approval under the FDA's expedited priority voucher program.
The U.S. Food and Drug Administration (FDA) announced on Friday, May 8, 2026, the approval of Bizengri (zenocutuzumab-zbco). This marks the first time a drug has been approved for adults suffering from advanced, inoperable, or metastatic cholangiocarcinoma that features an NRG1 gene fusion.
NRG1 gene fusions are an abnormality where DNA segments join, signaling cells to grow uncontrollably and leading to cancer. Cholangiocarcinoma, cancer of the bile ducts, has been notoriously difficult to treat, with few effective options available post-initial therapy.
Bizengri's approval follows its prior clearance in 2024 for specific types of lung and pancreatic cancers with similar genetic fusions. This latest clearance was granted under the Commissioner's National Priority Voucher (CNPV) pilot program, designed to accelerate the review of therapies for rare diseases.
Clinical trials involving 19 adults with NRG1 fusion-positive cholangiocarcinoma demonstrated a 37% response rate to Bizengri, with response durations varying from three to thirteen months. While offering hope, potential serious side effects include infusion reactions, lung issues, and heart dysfunction, alongside more common side effects like fatigue and digestive upset. The approval was awarded to Partner Therapeutics, Inc.
