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The US Food and Drug Administration (FDA) issued an approval for Bizengri (zenocutuzumab-zbco), a drug that treats NRG1 fusion-positive cholangiocarcinoma, an ultra-rare, aggressive cancer that forms in the bile ducts.
Bizengri is the first drug approved for adults with advanced, unresectable or metastatic cholangiocarcinoma harbouring a neuregulin 1 (NRG1) gene fusion with disease progression on or after prior systemic therapy.
This approval marks the seventh approval under the Commissioner’s National Priority Voucher (CNPV) pilot programme.
“Patients with this ultra-rare type of cancer desperately need new treatment options,” said FDA Commissioner Marty Makary, M.D., M.P.H. “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.
The efficacy of Bizengri was evaluated in a single-arm trial of 19 patients with NRG1 fusion-positive cholangiocarcinoma of which 36.8% had an overall response. The duration of response ranged from 2.8 months to 12.9 months.
The FDA granted Bizengri Breakthrough Therapy and Orphan Drug designations.
In 2024, Bizengri received accelerated approval for the treatment of adults with advanced unresectable or metastatic non-small cell lung cancer and pancreatic adenocarcinoma harbouring a NRG1 gene fusion with disease progression on or after prior systemic therapy.
Serious side effects associated with Bizengri include infusion-related reactions, interstitial lung disease/pneumonitis, and left ventricular dysfunction. The most common side effects include diarrhoea, musculoskeletal pain, fatigue, nausea, infusion-related reactions, dyspnoea, rash, constipation, vomiting, abdominal pain, and edema.
The FDA granted the approval to Partner Therapeutics, Inc.
On June 4, 2026, the FDA will host a public meeting to solicit feedback about the CNPV pilot program’s eligibility criteria, the voucher selection process, sponsor’s responsibilities, pre-submission requirements, FDA review procedures, the role of the CNPV review council, and programme implementation. Interested parties may also submit written comments through June 29, 2026.
The FDA, an agency within the US Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, radiation-emitting electronic products, and for regulating tobacco products.
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