Jazz Pharmaceuticals plc (NASDAQ: JAZZ) has recently announced the U.S. Food and Drug Administration (FDA) accelerated approval of ZIHERA® (zanidatamab-hrzg) 50mg/ml for injection for intravenous use for the treatment of adults with previously-treated, unresectable or metastatic HER2-positive (IHC 3+) biliary tract cancer (BTC), as detected by an FDA-approved test. This approval is based on compelling data from the HERIZON-BTC-01 trial, which included the evaluation of zanidatamab as a single agent in previously treated HER2-positive BTC and is the largest phase 2b clinical trial to date specifically for this patient population.
The efficacy of ZIHERA was evaluated in 62 patients with HER2-positive (IHC 3+ by central assessment) BTC in cohort 1 of HERIZON-BTC-01, with major efficacy outcome measures of objective response rate (ORR) and duration of response (DOR) as determined by independent central review (ICR). The study demonstrated an ORR of 52% and a Kaplan Meier estimated median DOR of 14.9 months by ICR.
The safety profile for ZIHERA has been demonstrated in 80 patients in the HERIZON-BTC-01 trial. Serious adverse reactions occurred in 53% of patients who received ZIHERA, with the most common adverse reactions being diarrhea, infusion-related reaction, abdominal pain, and fatigue.
Looking ahead, the confirmatory, global, randomized phase 3 trial HERIZON-BTC-302 (NCT06282575) is ongoing and evaluating zanidatamab in combination with standard-of-care therapy versus standard-of-care therapy alone in the first-line setting for patients with HER2-positive BTC. Continued approval for ZIHERA may be contingent upon verification and description of clinical benefit in this confirmatory trial.
Zanidatamab is also being investigated in a number of additional tumor types, including phase 3 trials in gastroesophageal adenocarcinomas (GEAs) and metastatic breast cancer (MBC).
The company will host an investor webcast on Wednesday, December 11, 2024, at 4:30 p.m. ET / 9:30 p.m. GMT to provide investors an overview of clinical data, patient need, and commercialization strategy for ZIHERA. The webcast will include commentary from a leading BTC expert and the company’s senior management.
ZIHERA is a bispecific HER2-directed antibody that binds to two extracellular sites on HER2, inducing complement-dependent cytotoxicity (CDC), antibody-dependent cellular cytotoxicity (ADCC), and antibody-dependent cellular phagocytosis (ADCP). In the United States, ZIHERA is indicated for the treatment of adults with previously treated, unresectable or metastatic HER2-positive (IHC 3+) biliary tract cancer (BTC), as detected by an FDA-approved test.
The FDA granted breakthrough therapy designation for zanidatamab development in patients with previously treated HER2 gene-amplified BTC and two fast track designations for zanidatamab: one as a single agent for refractory BTC and one in combination with standard-of-care chemotherapy for 1L gastroesophageal adenocarcinoma (GEA).
Jazz Pharmaceuticals plc is a global biopharma company whose purpose is to innovate to transform the lives of patients. Today the company's shares have moved 5.2% to a price of $125.48. Check out the company's full 8-K submission here.
