FDA approves Zenocutuzumab for NRG1 fusion-positive advanced cholangiocarcinoma

The U.S. Food and Drug Administration has granted approval for zenocutuzumab-zbco, marketed as Bizengri, to treat adult patients with NRG1 fusion‑positive advanced, unresectable or metastatic cholangiocarcinoma who have progressed after prior systemic therapy.

Approval was based on a single‑arm phase 1/2 eNRGy trial that evaluated 19 patients with the biomarker‑defined disease. The study reported an overall response rate of 36.8% as assessed by blinded independent central review.

Responses lasted between 2.8 and 12.9 months, indicating that a subset of patients may experience clinically meaningful benefit despite heavy pretreatment. The FDA used the national priority voucher pilot program to accelerate the review, reflecting the unmet medical need in this ultra‑rare cancer population.