The FDA has approved Bizengri (zenocutuzumab-zbco) for adults with advanced or metastatic NRG1 fusion-positive cholangiocarcinoma after prior systemic therapy, making it the first approved treatment for this rare bile duct cancer subtype.
Written By: Sana Khan, BPharm
Reviewed By: Pharmacally Editorial Team
The U.S. Food and Drug Administration (FDA) has approved Bizengri (zenocutuzumab-zbco) for adults with advanced, unresectable, or metastatic cholangiocarcinoma harboring a neuregulin 1 (NRG1) gene fusion whose disease has progressed on or after prior systemic therapy. This approval positions Bizengri as the first FDA-approved treatment specifically for this ultra-rare, aggressive bile duct cancer subtype.
Cholangiocarcinoma arises in the bile ducts, with NRG1 gene fusions representing rare genetic drivers of tumor growth. Patients with NRG1 fusion-positive disease face limited options post-standard therapy failure.
Approval stemmed from the eNRG1-002 single-arm trial (n=19 patients with NRG1 fusion-positive cholangiocarcinoma), yielding a 36.8% overall response rate (ORR). Median duration of response ranged from 2.8 to 12.9 months.
FDA Commissioner Marty Makary emphasized that patients with this ultra-rare cancer require more options, crediting the approval to the agency’s acceleration of rare disease therapies via the Commissioner’s National Priority Voucher (CNPV) pilot program. This marks the seventh CNPV authorization.
Bizengri earned prior accelerated approval in 2024 for NRG1 fusion-positive advanced unresectable or metastatic non-small cell lung cancer (NSCLC) and pancreatic adenocarcinoma after prior therapy progression. It also received Breakthrough Therapy and Orphan Drug designations for cholangiocarcinoma.
Serious adverse reactions include infusion-related reactions, interstitial lung disease/pneumonitis, and left ventricular dysfunction. Common adverse events (>20%) encompass diarrhea, musculoskeletal pain, fatigue, nausea, infusion-related reactions, dyspnea, rash, constipation, vomiting, abdominal pain, and edema.
Partner Therapeutics received approval. The FDA will host a public meeting on June 4, 2026, for CNPV feedback (eligibility, procedures, implementation); written comments are due by June 29, 2026.
Reference
FDA Grants Seventh Approval under the National Priority Voucher Pilot Program | FDA
About the Writer
Sana Jamil Khan (LinkedIn) is a B. Pharm graduate with a strong interest in medical writing and scientific communication. Her work focuses on interpreting clinical research, exploring developments in pharmaceutical science, and presenting complex medical information in a clear and accessible manner. She is particularly interested in topics related to human clinical studies, drug safety observations, and emerging therapeutic research.