National Comprehensive Cancer Network Adds Jazz’s Ziihera as a Category 2A Treatment Option for Biliary Tract Cancers

Image Credit: Adobe Stock Images/Sebastian Kaulitzki

The National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology have added Jazz Pharmaceuticals’ Ziihera (zanidatamab-hrii) as a category 2A treatment option for biliary tract cancers (BTC).1 The addition comes shortly after the FDA granted Ziihera accelerated approval late last month for the treatment of adults with previously treated, unresectable or metastatic HER2-positive (IHC 3+) BTC, as detected by an FDA-approved test.²

"The addition of Ziihera to the NCCN Guidelines in Oncology reaffirms the importance of this advancement in treating BTC and supports our commitment to ensuring that HER2-positive BTC patients, through their health care professionals, can access this important new therapeutic option," said Rob Iannone, MD, MSCE, EVP, global head, research and development, chief medical officer, Jazz Pharmaceuticals, in a press release. "Ziihera is the first and only dual HER2-targeted bispecific antibody and chemotherapy-free treatment for patients living with BTC – a disease with a poor prognosis and limited treatment options. We are excited to deliver this new treatment option to patients in the United States following its accelerated approval."¹

The FDA accelerated approval was based on results from the Phase IIb HERIZON-BTC-01 trial, which demonstrated that among the overall evaluable patient population enrolled in cohort 1 (n = 80), the confirmed objective response rate (cORR) among those administered Ziihera was 41.3%, with a disease control rate of 68.8%. Additionally, the cORR was 51.6% in a subgroup of patients with HER2-positive IHC 3+ disease and 5.6% in patients with IHC 2+ disease. Across a median follow-up of 22 months, median duration of response increased to 14.9 months.

Further, the data demonstrated that median overall survival (OS) in the overall population in cohort 1 was 15.5 months, whereas it was 18.1 months in patients with HER2-positive IHC 3+ disease, and 5.2 months in patients with IHC 2+ disease. Further, the one-year OS rates were 56.2% in the overall population, 65.0% in the IHC 3+ subgroup, and 20.8% in the IHC 2+ subgroup.²

"BTC is a devastating disease with a poor prognosis and five-year survival rates under 5% in the metastatic setting. Patients with unresectable or metastatic HER2-positive BTC have had a high unmet need with limited treatment options and few approved therapies," Rob Iannone, MD, MSCE, EVP, global head, research and development, chief medical officer, Jazz Pharmaceuticals, said in a press release. "The approval of Ziihera, which previously received Breakthrough Therapy Designation from the FDA for this indication, is an important advance and offers the first and only dual HER2-targeted bispecific antibody and chemotherapy-free treatment for patients living with BTC. We look forward to advancing research of zanidatamab in BTC and other HER2-expressing solid tumors, with the goal of improving outcomes for more people diagnosed with these difficult-to-treat HER2-positive cancers."

According to the American Cancer Society, bile duct cancer is rare in the United States, as approximately 8,000 people are currently living with the disease, including patients with intrahepatic and extrahepatic bile duct cancers. However, the exact numbers are unknown, as these cancers can be hard to diagnose, and some might be misclassified as other types of cancer. On average, it is most commonly diagnosed at 70 years of age.³

Jazz warns that treatment with Ziihera can result in left ventricular dysfunction, a number of injection-related reactions, and severe diarrhea. Other common adverse events include abdominal pain and fatigue.⁴

References

1. Jazz Pharmaceuticals Announces Update to National Comprehensive Cancer Network® (NCCN®) Clinical Practice Guidelines in Oncology for Biliary Tract Cancers to Include zanidatamab-hrii (Ziihera®). PR Newswire. December 5, 2024. Accessed December 6, 2024. https://www.prnewswire.com/news-releases/jazz-pharmaceuticals-announces-update-to-national-comprehensive-cancer-network-nccn-clinical-practice-guidelines-in-oncology-for-biliary-tract-cancers-to-include-zanidatamab-hrii-ziihera-302323285.html

2. FDA Grants Accelerated Approval to Jazz Pharmaceuticals’ Ziihera for HER2-Positive Biliary Tract Cancer. PharmExec. November 21, 2024. Accessed December 6, 2024. https://www.pharmexec.com/view/fda-accelerated-approval-jazz-pharmaceuticals-ziihera-her2-biliary-tract-cancer

3. Key Statistics for Bile Duct Cancer. American Cancer Society. Accessed December 6, 2024. https://www.cancer.org/cancer/types/bile-duct-cancer/about/key-statistics.html

4. HIGHLIGHTS OF PRESCRIBING INFORMATION. Jazz. Accessed December 6, 2024. https://pp.jazzpharma.com/pi/ziihera.en.USPI.pdf