Jazz Pharmaceuticals Gains U.S. FDA Approval for Ziihera® in HER2-Positive Biliary Tract Cancer
Thursday, November 21, 2024
Jazz Pharmaceuticals has announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Ziihera® (zanidatamab-hrii), a treatment for adults with previously treated, unresectable, or metastatic HER2-positive biliary tract cancer (BTC).
BTC is a rare and aggressive form of cancer with limited treatment options, particularly in its advanced stages. Ziihera is the first chemotherapy-free, bispecific antibody targeting HER2 for this patient group.
The pivotal HERIZON-BTC-01 trial evaluated the efficacy and safety of Ziihera in 62 patients with HER2-positive BTC. Results confirmed its antitumour activity, with a significant proportion of patients responding positively to treatment.
The approval is specifically for cancers confirmed as HER2-positive (IHC 3+) using an FDA-approved diagnostic test.
The decision was based on data from the HERIZON-BTC-01 trial, which demonstrated an objective response rate (ORR) of 52% and a median duration of response (DOR) of 14.9 months.
The FDA's accelerated approval programme allows for earlier access to treatments showing promising results, with continued approval subject to further evidence from confirmatory trials.
Additional studies are underway to confirm Ziihera's clinical benefits. The Phase 3 HERIZON-BTC-302 trial is assessing the drug in combination with standard therapies as a first-line treatment for HER2-positive BTC.
The safety of Ziihera was evaluated in the Phase 2b HERIZON-BTC-01 trial, which involved 80 patients. Serious adverse reactions were reported in 53% of participants, with the most common being biliary obstruction, infections, and sepsis.
Other frequent side effects included diarrhoea, fatigue, and abdominal pain. A boxed warning highlights the potential risk of embryo-fetal toxicity, necessitating the use of effective contraception for patients of reproductive age.
Ziihera is a bispecific HER2-directed antibody that binds to two extracellular regions of the HER2 protein, reducing its presence on tumour cells and triggering multiple mechanisms to inhibit tumour growth and induce cell death.
Beyond BTC, Ziihera is being explored in other HER2-positive cancers, including gastroesophageal adenocarcinomas and metastatic breast cancer, through various clinical trials. These studies aim to establish its potential in combination therapies and expand treatment options for patients with HER2-driven malignancies.
This approval marks a significant advancement in the management of HER2-positive BTC, addressing an unmet medical need and offering hope to patients affected by this challenging condition.
Source: jazzpharma.com
