WASHINGTON, D.C. — The U.S. Food and Drug Administration has approved Bizengri for adults with advanced NRG1 fusion-positive cholangiocarcinoma, marking the first approved treatment for the rare bile duct cancer after progression on prior systemic therapy.
The approval covers patients with unresectable or metastatic cholangiocarcinoma harboring a neuregulin 1 gene fusion. The FDA said the decision was made under the agency’s Commissioner’s National Priority Voucher pilot program, which is designed to accelerate reviews for therapies targeting rare diseases and unmet medical needs.
Marty Makary said patients with the ultra-rare cancer have limited treatment options.
“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,” Makary said.
The FDA based its decision on data from a single-arm clinical trial involving 19 patients with NRG1 fusion-positive cholangiocarcinoma. According to the agency, 36.8% of patients experienced an overall response to treatment. Duration of response ranged from 2.8 months to 12.9 months.
The agency granted Bizengri both Breakthrough Therapy and Orphan Drug designations.
Bizengri previously received accelerated FDA approval in 2024 for adults with advanced unresectable or metastatic non-small cell lung cancer and pancreatic adenocarcinoma with NRG1 gene fusions following prior systemic therapy.
The FDA said serious risks associated with the drug include infusion-related reactions, interstitial lung disease or pneumonitis, and left ventricular dysfunction. Common side effects reported during testing included diarrhea, musculoskeletal pain, fatigue, nausea, dyspnea, rash, constipation, vomiting, abdominal pain and edema.
The approval was granted to Partner Therapeutics.
The FDA also said it will hold a public meeting on June 4, 2026, to gather feedback on the Commissioner’s National Priority Voucher pilot program, including eligibility standards, review procedures and implementation. Written comments will be accepted through June 29, 2026.
Support the local news that supports Chester County. MyChesCo delivers reliable, fact-based reporting and essential community resources—free for everyone. If you value that, click here to become a patron today.