Jazz Pharmaceuticals (JAZZ) Status Update summary

Regulatory and Clinical Milestones

• Zanidatamab (Ziihera) received accelerated FDA approval as the first dual HER2-targeted bispecific antibody for previously treated unresectable or metastatic HER2-positive BTC in adults in the U.S.

• Approval was based on the HERIZON-BTC-01 phase IIb trial, which showed a confirmed overall response rate (ORR) of 41.3% and a median duration of response of 14.9 months in HER2-positive BTC patients, with higher efficacy in IHC3+ patients (ORR 51.6%).

• Median overall survival reached 15.5 months, with 18.1 months in IHC3+ patients.

• Ziihera is now available to prescribers in the U.S. and is included in NCCN Guidelines and Treatment Compendia.

• Safety profile was manageable, with serious or high-grade treatment-related adverse events in 9.2% and no treatment-related deaths.

Clinical Data and Mechanism of Action

• The HERIZON-BTC-01 trial enrolled patients with HER2-amplified BTC who had progressed after gemcitabine-based chemotherapy; most had stage IV disease and IHC3+ status.

• Zanidatamab binds two non-overlapping HER2 domains, inducing receptor clustering, complement-dependent cytotoxicity, and immune-mediated effects, differentiating it from other HER2 therapies.

• Preclinical and clinical data show greater activity than trastuzumab ± pertuzumab, with strong activation of immune effector functions.

Market Opportunity and Commercial Launch

• Ziihera’s launch targets approximately 3,000 annual U.S. patients with HER2-expressing BTC, addressing a high unmet need and poor prognosis.

• The wholesale acquisition cost is $7,110 per two-pack (300mg vials), with a 28-day cycle cost of about $35,500.

• Ziihera’s dual-targeting mechanism provides a differentiated, chemotherapy-free treatment with improved quality of life.

• Jazz aims to establish Ziihera as the standard of care for 2L HER2+ BTC, supported by a proven oncology team and robust patient support infrastructure.