FDA Approves Zenocutuzumab for Patients With NRG1 Fusion-Positive Advanced Cholangiocarcinoma
Clinical Summary:
- Based on results from the phase 2 eNRGy trial, the FDA has approved zenocutuzumab for previously treated patients with advanced, unresectable, or metastatic cholangiocarcinoma harboring NRG1 gene fusions.
- In this multicenter, open-label study, zenocutuzumab demonstrated objective responses with durable activity, with a manageable safety profile consistent with targeted biologic therapy.
- This approval provides a targeted treatment option for patients with NRG1 fusion-positive cholangiocarcinoma, a rare population with limited therapeutic options.
On May 8, 2026, the US Food and Drug Administration (FDA) approved zenocutuzumab (Bizengri; Partner Therapeutics, Inc.) for adult patients with advanced, unresectable, or metastatic cholangiocarcinoma harboring neuregulin 1 (NRG1) gene fusions who have experienced disease progression on or after prior systemic therapy. This approval was based on results from the phase 2 eNRGy trial.
In this multicenter, open-label study, 19 patients with NRG1 fusion-positive cholangiocarcinoma received 750 mg of zenocutuzumab once every 2 weeks until disease progression or unacceptable toxicity. Primary end points included confirmed objective response rate (ORR) and duration of response, as assessed by blinded independent central review.
At analysis, the ORR was 36.8%, with duration of responses lasting from 2.8 to 12.9 months. The most frequently reported adverse events included diarrhea, musculoskeletal pain, fatigue, nausea, infusion-related reactions, dyspnea, rash, constipation, vomiting, abdominal pain, and edema.
The recommended dose of zenocutuzumab is 750 mg administered as an intravenous infusion every 2 weeks until disease progression or unacceptable toxicity. Zenocutuzumab includes warnings for infusion-related reactions, hypersensitivity including anaphylaxis, interstitial lung disease/pneumonitis, left ventricular dysfunction, and embryo-fetal toxicity.
Sources:
US Food and Drug Administration. FDA approves zenocutuzumab-zbco for advanced, unresectable or metastatic cholangiocarcinoma. Accessed on May 8, 2026. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-zenocutuzumab-zbco-advanced-unresectable-or-metastatic-cholangiocarcinoma
Schram AM, Goto K, Kim DW, et al. Efficacy of zenocutuzumab in NRG1 fusion–positive cancer. Published online: February 5, 2025. doi: 10.1056/NEJMoa2405008
