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FDA Approves Bizengri for NRG1 Fusion–Positive Cholangiocarcinoma

The decision uses a priority voucher program to speed a targeted option for a small group with few treatments.

Signage is seen outside of the Food and Drug Administration (FDA) headquarters in White Oak, Maryland, U.S., August 29, 2020. REUTERS/Andrew Kelly/File Photo

Overview

  • The FDA approved zenocutuzumab-zbco (Bizengri) for adults with advanced, unresectable or metastatic cholangiocarcinoma that harbors NRG1 fusions after prior systemic therapy.
  • Regulators based the decision on the single-arm eNRGy study, where 19 evaluable patients had a 36.8% overall response rate with responses lasting 2.8 to 12.9 months.
  • The label warns about infusion reactions, interstitial lung disease or pneumonitis, and left ventricular dysfunction, and it lists diarrhea, fatigue, nausea, dyspnea, rash, constipation, vomiting, abdominal pain, edema, and musculoskeletal pain as common side effects.
  • The approval is the seventh granted under the FDA’s National Priority Voucher pilot program, which shortens reviews for therapies that address rare diseases with unmet needs.
  • The multicenter, open-label eNRGy trial enrolled 22 patients with this cancer subtype and offers a targeted option for an ultra-rare driver where few effective treatments exist after first-line care.