Partner Therapeutics, Inc. (PTx), a private, fully integrated biotechnology company, today announced that the U.S. Food and Drug Administration (FDA) has awarded a Commissioner’s National Priority Voucher (CNPV) pilot program voucher for BIZENGRI® (zenocutuzumab-zbco) for the treatment of adults with advanced, unresectable or metastatic cholangiocarcinoma harboring a neuregulin 1 (NRG1) gene fusion with disease progression on or after prior systemic therapy. PTx submitted a supplemental Biologics License Application (sBLA) to the FDA for this indication based on data from the Phase 2 eNRGy trial. BIZENGRI previously received Breakthrough Therapy Designation and Orphan Drug Designation from the FDA for NRG1+ cholangiocarcinoma.
“We are honored to receive this Commissioner’s National Priority Voucher, which reflects the FDA’s recognition of the profound unmet need facing patients with NRG1+ cholangiocarcinoma, an ultra-rare cancer for which no approved targeted therapy currently exists,” said Pritesh J. Gandhi, Chief Development Officer, Partner Therapeutics. “Receiving this voucher highlights the urgent need for new treatment options in NRG1 fusion-positive cholangiocarcinoma. Based on the encouraging data from the eNRGy trial, including meaningful tumor responses, durable benefit and a favorable tolerability profile, we believe BIZENGRI has the potential to address a critical gap in care for these patients. We look forward to working closely with the FDA through this accelerated review process.”
The FDA’s Commissioner’s National Priority Voucher pilot program, announced in June 2025, is designed to reduce drug and biologic review times from the standard 10–12 months to as little as 1–2 months for products that align with one or more U.S. national health priorities. These priorities focus on advancing innovative breakthrough therapies that introduce novel mechanisms and meaningfully improve how diseases are treated, while also addressing significant unmet medical needs where current treatment options fall short for patients. The program employs a multidisciplinary, tumor board-style review process involving the primary FDA review team and senior agency leadership. BIZENGRI, as a first-in-class bispecific antibody targeting a rare and actionable oncogenic driver with no approved targeted therapy, aligns directly with national priorities.
“For patients facing cholangiocarcinoma, innovation and timely diagnosis can make a profound difference. The FDA’s recognition of this potential treatment underscores both the unmet need in this rare biomarker-defined population and the importance of expanding access to comprehensive biomarker testing so patients can be matched to the most appropriate therapies as early as possible,” said Stacie Lindsey, CEO of the Cholangiocarcinoma Foundation.
Data from the cohort of patients with NRG1+ cholangiocarcinoma in the Phase 2 eNRGy trial were presented at the AACR-NCI-EORTC Meeting in October 2025, highlighting clinical outcomes in this rare cancer.1 “Patients with NRG1 fusion–positive cholangiocarcinoma face a significant unmet need, with limited effective treatment options available today,” said Alison Schram, MD, Gynecologic Medical Oncologist at Memorial Sloan Kettering Cancer Center (MSK) and Principal Investigator of the eNRGy trial. “The FDA’s recognition through the Commissioner’s National Priority Voucher program emphasizes the urgency of advancing therapies for this population. In the eNRGy study, zenocutuzumab demonstrated clinically meaningful activity, with objective responses in more than one-third of evaluable patients and a median progression-free survival of over nine months. These data also highlight the critical role of comprehensive molecular testing, particularly RNA-based comprehensive molecular profiling, in identifying patients who may benefit from emerging targeted approaches.”
About NRG1+ Cholangiocarcinoma
Cholangiocarcinoma is a rare, aggressive malignancy of the bile ducts with an all-stage 5-year overall survival of less than 15%. NRG1 gene fusions occur in fewer than 1% of cholangiocarcinoma cases. NRG1 fusions are largely mutually exclusive with other actionable oncogenic drivers, leaving affected patients—many of whom are younger adults —without approved targeted therapy. Standard cytotoxic regimens carry substantial toxicity, and second-line options such as FOLFOX produce objective responses in only approximately 5% of patients.
