MONDAY, May 11, 2026 (HealthDay News) -- The U.S. Food and Drug Administration has approved Bizengri (zenocutuzumab-zbco) for the treatment of neuregulin 1 (NRG1) fusion-positive cholangiocarcinoma.
This approval occurred under the National Priority Voucher pilot program and also received Breakthrough Therapy and Orphan Drug designations. Bizengri is the first drug approved for adults with advanced, unresectable, or metastatic cholangiocarcinoma harboring an NRG1 gene fusion with disease progression on or after prior systemic therapy.
The approval was based on data from a single-arm trial of 19 patients with NRG1 fusion-positive cholangiocarcinoma, of whom 36.8 percent had an overall response, ranging in duration from 2.8 to 12.9 months. Serious side effects included infusion-related reactions, interstitial lung disease/pneumonitis, and left ventricular dysfunction, as well as common side effects of diarrhea, musculoskeletal pain, fatigue, nausea, infusion-related reactions, dyspnea, rash, constipation, vomiting, abdominal pain, and edema.
"Patients with this ultra-rare type of cancer desperately need new treatment options," FDA Commissioner Marty Makary, M.D., said in a statement. "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."
Approval of Bizengri was granted to Partner Therapeutics.
