Jazz completes rolling submission of BLA for zanidatamab antibody

Jazz Pharmaceuticals announced that the company has completed the rolling submission of a Biologics License Application, or BLA, to FDA, seeking accelerated approval for the HER2-targeted bispecific antibody zanidatamab as a treatment for previously-treated, unresectable, locally advanced, or metastatic HER2-positive biliary tract cancer, or BTC. If approved, zanidatamab would be the first HER2-targeted treatment specifically approved for BTC in the U.S. The BLA includes data from the Phase 2b HERIZON-BTC-01 trial of zanidatamab in previously treated HER2-positive BTC. The primary endpoint was confirmed objective response rate, or cORR, by independent central review, or ICR, in Cohort 1. Data as of Oct. 10, 2022, from the 80 HER2-positive BTC patients enrolled in Cohort 1 of the trial demonstrated a cORR of 41.3% with a Kaplan Meier, or KM, estimated median duration of response, or DOR, of 12.9 months by ICR. Historical response rates for 2L standard-of-care chemotherapy in patients with BTC are reported to be 5 to 15%1,2. The KM estimated median progression-free survival, or PFS, was 5.5 months with a range of 0.3 to 18.5 months. Zanidatamab demonstrated a manageable and tolerable safety profile, with only two patients, or 2.3%, in HERIZON-BTC-01 experiencing adverse events (AEs) leading to treatment discontinuation. There were no Grade 4 AEs, and no deaths were considered treatment-related. The most common AEs were diarrhea and infusion-related reactions, which were predominately low-grade, reversible and manageable prophylactically with routine supportive care. These data were featured as an oral presentation at the American Society of Clinical Oncology Annual Meeting 2023, published in The Lancet Oncology, and included in the 2023 Best of ASCO program.