The US Food and Drug Administration (FDA) has approved Bizengri (zenocutuzumab) for the treatment of adults with NRG1 fusion-positive cholangiocarcinoma.
Cholangiocarcinoma is a rare, aggressive cancer that forms in the bile duct. Patients with cholangiocarcinoma are often diagnosed at a later stage when the prognosis is poor. The disease can be classified based on whether the cancer is in the bile duct within the liver (intrahepatic cholangiocarcinoma [iCCA]) or outside the liver (extrahepatic cholangiocarcinoma). NRG1 gene fusions occur in less than 1% of cases and typically occur in patients who are otherwise driver negative, leaving affected patients without approved targeted therapy.
The approval is based on safety and efficacy data from the phase 2 eNRGy clinical trial, a multicenter, open-label, multi-cohort study in adults with advanced solid tumors with NRG1 gene fusions. A total of 22 patients with unresectable or metastatic NRG1 fusion-positive cholangiocarcinoma were enrolled, with 19 evaluable for efficacy.
The major efficacy outcome measures were confirmed overall response rate (ORR), which is the percentage of patients in a clinical trial whose cancer shrinks or disappears after treatment, and duration of response (DOR). The OOR was 36.8% with a DOR of 2.8 to 12.9 months.
Serious side effects associated with zenocutuzumab include infusion-related reactions, interstitial lung disease/pneumonitis, and left ventricular dysfunction. The most common adverse reactions were fatigue, diarrhea, musculoskeletal pain, abdominal pain, nausea, cough, dyspnea, and decreased appetite.
The approval was expedited by a National Priority Voucher (CNPV), only the seventh approval under this FDA pilot program. The program seeks to expedite approval of applications that address critical national health priorities, such as bringing innovative therapies to the American people, addressing large unmet medical needs, promoting domestic manufacturing, and increasing affordability. A company selected for the program is issued a voucher entitling the company to benefits including enhanced communications and rolling review to allow for a shortened review time.
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To learn more about cholangiocarcinoma and other rare cancers, visit https://checkrare.com/diseases/cancers/