Jazz Pharmaceuticals (Nasdaq: JAZZ) has announced that the U.S. Food and Drug Administration (FDA) granted accelerated approval for Ziihera® (zanidatamab-hrii) 50mg/mL injection. This intravenous treatment is for adults with previously treated, unresectable, or metastatic HER2-positive biliary tract cancer (BTC). Approval is based on a 52% objective response rate (ORR) and a median duration of response (DOR) of 14.9 months observed in clinical trials. Continued approval is contingent on results from the ongoing Phase 3 HERIZON-BTC-302 trial, which evaluates Ziihera in combination with standard care.
BTC is a highly aggressive cancer with a five-year survival rate of less than 5% in metastatic cases. Rob Iannone, M.D., Chief Medical Officer at Jazz Pharmaceuticals, highlighted the breakthrough significance of Ziihera, the first dual HER2-targeted, chemotherapy-free treatment for this condition.
Ziihera’s FDA approval stems from the HERIZON-BTC-01 Phase 2b trial, which reported compelling results presented at ASCO and published in The Lancet Oncology. The study achieved a confirmed ORR of 52% and a median DOR of 14.9 months.
Expert and Advocacy Perspectives
Dr. James Harding of Memorial Sloan Kettering Cancer Center underscored the new hope Ziihera brings to patients with limited treatment options, while Stacie Lindsey, CEO of the Cholangiocarcinoma Foundation, praised its potential to improve quality of life for BTC patients and their families.
Safety and Warnings
Common side effects included diarrhea, infusion-related reactions, abdominal pain, and fatigue, with serious adverse events reported in 53% of patients. There is a boxed warning for embryo-fetal toxicity, emphasizing the need for effective contraception.
Future Research
Ziihera is being studied in other HER2-positive cancers, including gastroesophageal adenocarcinomas and metastatic breast cancer, through trials such as HERIZON-GEA-01 and EmpowHER-303.
