FDA Grants Breakthrough Therapy Status to Zenocutuzumab-zbco for NRG1+ Cancer
Partner Therapeutics, Inc. (PTx) announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to zenocutuzumab-zbco for the treatment of adults with advanced unresectable or metastatic cholangiocarcinoma harbouring a neuregulin 1 (NRG1) gene fusion.
The designation is based on encouraging data from the ongoing Phase 2 eNRGy trial, with results presented at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in Boston.
In the eNRGy trial, zenocutuzumab-zbco demonstrated an investigator-assessed overall response rate of 37%, a median duration of response of 7.4 months, and a clinical benefit rate of 58% in patients with NRG1+ cholangiocarcinoma.
The treatment showed a manageable safety profile, with most adverse events reported as mild or moderate.
The Breakthrough Therapy Designation aims to expedite the development and review of promising treatments for serious or life-threatening conditions. It provides enhanced FDA guidance and eligibility for priority review.
Zenocutuzumab-zbco, marketed as BIZENGRI, previously received accelerated approval in December 2024 for adults with advanced NRG1+ non-small cell lung cancer (NSCLC) and pancreatic adenocarcinoma.
Partner Therapeutics plans to submit a supplemental Biologics License Application (sBLA) for cholangiocarcinoma to the FDA in 2026.
