The U.S. Food and Drug Administration (FDA) has approved Bizengri (zenocutuzumab-zbco) for the treatment of adults with advanced, unresectable or metastatic cholangiocarcinoma harboring a neuregulin 1 (NRG1) gene fusion, who have experienced disease progression following prior systemic therapy. This marks the seventh drug approval under the FDA Commissioner’s National Priority Voucher (CNPV) pilot program.
Cholangiocarcinoma is an aggressive and ultra-rare form of cancer that originates in the bile ducts. Bizengri is the first therapy approved specifically for patients with NRG1 fusion-positive cholangiocarcinoma. The approval was granted to Partner Therapeutics, Inc.
Clinical evidence supporting approval
The FDA based its decision on results from a single-arm clinical trial involving 19 patients with NRG1 fusion-positive cholangiocarcinoma. The overall response rate observed was 36.8%, with the duration of response ranging from 2.8 to 12.9 months. Bizengri has previously received Breakthrough Therapy and Orphan Drug designations from the FDA.
In 2024, the FDA also granted accelerated approval for Bizengri in treating adults with advanced, unresectable, or metastatic non-small cell lung cancer and pancreatic adenocarcinoma bearing an NRG1 gene fusion, reflecting its broader potential for NRG1-driven cancers.
Safety profile
Serious side effects associated with Bizengri include infusion-related reactions, interstitial lung disease or pneumonitis, and left ventricular dysfunction. Common adverse reactions reported during clinical trials include diarrhea, musculoskeletal pain, fatigue, nausea, dyspnea, rash, constipation, vomiting, abdominal pain, and edema.
National Priority Voucher Pilot Program
The approval is part of the FDA’s National Priority Voucher pilot program, designed to expedite review of treatments addressing rare diseases with high unmet medical needs. This program aims to accelerate access to therapies for conditions affecting smaller patient populations by shortening regulatory timelines.
On June 4, 2026, the FDA will hold a public meeting to gather feedback on the CNPV program’s eligibility, voucher selection process, sponsor responsibilities, and review procedures. Written comments will be accepted through June 29, 2026.
Why it matters
This approval offers a new targeted treatment option for patients with an ultra-rare, difficult-to-treat cancer subtype, addressing a critical unmet need. The use of the National Priority Voucher pilot program demonstrates the FDA’s commitment to accelerating development and availability of therapies for rare diseases.
Sources
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