Jazz Pharmaceuticals has received accelerated approval from the U.S. Food and Drug Administration (FDA) for Ziihera (zanidatamab), a HER2-targeted bispecific antibody, for the treatment of adults with previously treated, unresectable, or metastatic HER2-positive biliary tract cancer (BTC). This approval marks a significant advancement in the treatment landscape for BTC, a cancer with poor prognosis and limited therapeutic options.
Clinical Efficacy and Trial Data
The FDA's decision was supported by data from the HERIZON-BTC-01 trial, a study that demonstrated a 52% objective response rate (ORR) in patients treated with Ziihera. The median duration of response was 14.9 months, indicating a sustained benefit for responding patients. These results highlight Ziihera's potential to address the unmet needs of patients with HER2-positive BTC who have progressed on prior therapies.
Ziihera: A Novel HER2-Targeted Bispecific Antibody
Ziihera is designed to simultaneously bind to two different epitopes of the HER2 receptor, leading to enhanced receptor clustering, internalization, and subsequent tumor cell death. This dual-targeting mechanism differentiates Ziihera from traditional HER2-targeted therapies and may contribute to its observed clinical activity.
Addressing Unmet Needs in Biliary Tract Cancer
Biliary tract cancer is a rare and aggressive malignancy arising from the bile ducts. Patients with unresectable or metastatic HER2-positive BTC face a particularly poor prognosis, with five-year survival rates under 5%. Current treatment options are limited, often involving chemotherapy and, more recently, immunotherapies. Ziihera offers a new chemotherapy-free option for these patients.
Rob Iannone, head of R&D at Jazz Pharmaceuticals, stated, "The approval of Ziihera...is an important advance and offers the first and only dual HER2-targeted bispecific antibody and chemotherapy-free treatment for patients living with BTC."
Pricing and Market Considerations
Ziihera will be introduced with a list price of $7,110 for two 300 mg vials, translating to approximately $35,500 for a 28-day treatment cycle. The treatment is targeted towards a U.S. patient population estimated at around 3,000 individuals with first-line (1L) and second-line (2L) HER2+ BTC.
Expansion into Other HER2-Positive Cancers
Jazz Pharmaceuticals is actively investigating Ziihera's potential in other HER2-positive cancers, including gastroesophageal adenocarcinoma (GEA) and breast cancer. Phase 3 trials are underway in GEA, with results expected in the second quarter of next year. These studies could broaden Ziihera's applicability and establish it as a key player in the HER2-targeted therapy landscape.
Financial Implications for Jazz Pharmaceuticals
Analysts at Piper Sandler have reaffirmed an Overweight rating for Jazz Pharmaceuticals' stock, with a price target of $163.00, citing the FDA approval of Ziihera as a positive catalyst. While the BTC market is relatively small, the approval adds to the company's growth in the oncology segment. Jazz Pharmaceuticals' strong financial position, characterized by a healthy free cash flow yield and robust current ratio, supports continued portfolio expansion.
