FDA Alert

FDA Approves Zenocutuzumab-Zbco (Bizengri) for NRG1 Fusion-Positive Cholangiocarcinoma in Adults

Edited by:
Anthony Calabro, MA

Key Highlights

  • Bizengri (zenocutuzumab-zbco) is the first FDA-approved drug for adults with advanced, unresectable, or metastatic cholangiocarcinoma harboring an NRG1 gene fusion after disease progression on or after prior systemic therapy.
  • The approval was supported by a single-arm trial of 19 patients, in which 36.8% had an overall response.
  • Serious adverse events include infusion-related reactions, interstitial lung disease/pneumonitis, and left ventricular dysfunction.
  • The approval is the seventh issued under the Commissioner’s National Priority Voucher pilot program.

On May 8, the FDA approved zenocutuzumab-zbco (Bizengri) for adults with advanced, unresectable, or metastatic cholangiocarcinoma harboring a neuregulin 1 (NRG1) gene fusion with disease progression on or after prior systemic therapy. Zenocutuzumab-zbco is the first drug approved for this population. Cholangiocarcinoma is an aggressive cancer that forms in the bile ducts, and the FDA describes NRG1 fusion-positive disease as ultra-rare.

The approval was based on efficacy findings from a single-arm trial that enrolled 19 patients with NRG1 fusion-positive cholangiocarcinoma. In the trial, 36.8% of patients had an overall response. The duration of response ranged from 2.8 months to 12.9 months. The FDA granted zenocutuzumab-zbco Breakthrough Therapy and Orphan Drug designations.

“Patients with this ultra-rare type of cancer desperately need new treatment options,” Marty Makary, MD, MPH, said in a press release. “Through the national priority voucher pilot program, the FDA is accelerating therapies for rare diseases with unmet medical needs, reviewing applications in significantly shortened timelines.”

Serious side effects associated with zenocutuzumab-zbco include infusion-related reactions, interstitial lung disease/pneumonitis, and left ventricular dysfunction. The most common adverse events include diarrhea, musculoskeletal pain, fatigue, nausea, infusion-related reactions, dyspnea, rash, constipation, vomiting, abdominal pain, and edema.

Reference
US Food and Drug Administration. FDA grants seventh approval under the National Priority Voucher Pilot Program. Published May 8, 2026. Accessed May 12, 2026. https://www.fda.gov/news-events/press-announcements/fda-grants-seventh-approval-under-national-priority-voucher-pilot-program