<![CDATA[FDA Approves Zenocutuzumab for NRG1 Fusion+ Cholangiocarcinoma ]]>

Advertisement News|Articles|May 8, 2026 FDA Approves Zenocutuzumab for NRG1 Fusion+ Cholangiocarcinoma Author(s)Ashling Wahner Listen0:00 / 0:00 Key Takeaways Zenocutuzumab-zbco is now indicated for adult NRG1 fusion–positive advanced, unresectable, or metastatic cholangiocarcinoma following progression on or after prior systemic therapy. Single-arm eNRGy trial data in 19 evaluable patients demonstrated a 36.8% ORR, establishing antitumor activity in an ultra-rare, biomarker-defined cholangiocarcinoma population. Duration of response ranged from 2.8 to 12.9 months, suggesting variable but potentially meaningful clinical benefit in responders within this heavily pretreated setting. The FDA leveraged the national priority voucher pilot program to accelerate assessment of therapies targeting rare diseases with substantial unmet medical need.SHOW MORE Advertisement The FDA has approved zenocutuzumab-zbco (Bizengri) for the treatment of adult patients with NRG1 fusion–positive advanced, unresectable, or metastatic cholangiocarcinoma (CCA) who have experienced disease progression on or after prior systemic therapy.1,2This regulatory decision was supported by findings from the single-arm phase 1/2 eNRGy trial (NCT02912949), in which evaluable patients with NRG1 fusion–positive CCA (n = 19) achieved an overall response rate (ORR) of 36.8% (95% CI, 16.3%-61.6%) per blinded independent central review (BICR). Additionally, the duration of response ranged from 2.8 months to 12.9 months per BICR. “Patients with this ultra-rare type of cancer desperately need new treatment options,” FDA Commissioner Marty Makary, MD, MPH, stated in a news 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.” What was the design of the eNRGy trial? This multicenter, open-label, multicohort trial enrolled adult patients with advanced solid tumors, including 22 patients with unresectable or metastatic NRG1 fusion–positive CCA. Patients were required to have at least 1 measurable lesion per RECIST 1.1 criteria, an ECOG performance status of 0 to 2, and an estimated life expectancy of at least 12 weeks.3 Additionally, patients needed to have recovered from major surgery or other complications to grade 2 or less at baseline, have a maximum absolute neutrophil count of 1.5 x 109/L without colony stimulating factor support for at least 7 days before screening, have a minimum platelet counts of 75 x 109/L without transfusion support for at least 7 days before screening, and have hemoglobin levels of at least 8 g/dL or 5 mmol/L.Patients in the part 2 solid tumor basket portion of the study, including those with CCA, received intravenous zenocutuzumab at the recommended phase 2 dose of 750 mg every 2 weeks. ORR and DOR according to RECIST 1.1 criteria served as the primary end points. Secondary end points included CBR per BICR, time to response, progression-free survival, overall survival, and safety and tolerability. What is the safety profile of zenocutuzumab? Serious adverse effects (AEs) associated with zenocutuzumab include infusion-related reactions (IRRs), interstitial lung disease/pneumonitis, and left ventricular dysfunction. The most common AEs associated with this agent include diarrhea, musculoskeletal pain, fatigue, nausea, IRRs, dyspnea, rash, constipation, vomiting, abdominal pain, and edema.References FDA grants seventh approval under the National Priority Voucher Pilot Program. FDA. May 8, 2026. Accessed May 8, 2026. https://www.fda.gov/news-events/press-announcements/fda-grants-seventh-approval-under-national-priority-voucher-pilot-program?utm_medium=email&utm_source=govdeliveryFDA approves zenocutuzumab-zbco for advanced, unresectable or metastatic cholangiocarcinoma. FDA. May 8, 2026. Accessed May 8, 2026. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-zenocutuzumab-zbco-advanced-unresectable-or-metastatic-cholangiocarcinomaA study of zenocutuzumab (MCLA-128) in patients with solid tumors harboring an NRG1 fusion (eNRGy). ClinicalTrials.gov. Updated April 29, 2025. Accessed May 8, 2026. https://clinicaltrials.gov/study/NCT02912949 NewsletterStay up to date on the most recent and practice-changing oncology dataSubscribe Now! 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