Target Action (PDUFA) Date set for
November 29, 2024
If approved, zanidatamab will be the first
HER2-targeted treatment specifically indicated for patients with
HER2+ locally advanced or metastatic biliary tract cancer
DUBLIN, May 29, 2024 /PRNewswire/ -- Jazz
Pharmaceuticals plc (Nasdaq: JAZZ) today announced that the
U.S. Food and Drug Administration (FDA) has accepted and granted
Priority Review of the Biologics License Application (BLA) for
zanidatamab, the human epidermal growth factor receptor 2
(HER2)-targeted bispecific antibody, for the treatment of
previously treated, unresectable, locally advanced, or metastatic
HER2-positive biliary tract cancer (BTC). Under the
Prescription Drug User Fee Act (PDUFA), FDA has set a target action
date of November 29,
2024.
"The priority review designation for zanidatamab underscores the
critical need for new treatment options for patients with locally
advanced or metastatic HER2-positive BTC, a devastating disease
with a poor prognosis," said Rob
Iannone, M.D., M.S.C.E., executive vice president, global
head of research and development of Jazz Pharmaceuticals. "Upon
approval, zanidatamab will be the first HER2-targeted treatment
specifically indicated for these patients, and we look forward to
the opportunity to deliver this new treatment option to the BTC
community."
Jazz's BLA submission is based on results from Cohort 1 of
the Phase 2b HERIZON-BTC-01
clinical trial (NCT05152147) of zanidatamab in previously treated
patients with unresectable, locally advanced, or metastatic
HER2-positive BTC (defined as in situ hybridization [ISH] positive
and immunohistochemistry [IHC] 2+ or 3+). The trial demonstrated a
primary endpoint of 41.3% [95% confidence interval (CI): 30.4,
52.8] confirmed objective response rate (cORR) by independent
central review (ICR) and results were presented at the American
Society of Clinical Oncology (ASCO) Annual Meeting
2023, published in The Lancet Oncology, and
included in the 2023 Best of ASCO® program.
Overall survival, updated duration of response and additional
long-term follow-up data from the Phase 2b HERIZON-BTC-01 trial will be presented at the
upcoming ASCO Annual Meeting 2024.
Additionally, the global, open-label, randomized
HERIZON-BTC-302 Phase 3 trial (NCT06282575) to evaluate
the efficacy and safety of zanidatamab in combination with
standard-of-care therapy against standard-of-care therapy alone in
first-line advanced or metastatic HER2-positive BTC is ongoing and
is open for enrollment. HERIZON-BTC-302 is expected to serve as the
confirmatory trial for zanidatamab in BTC.
About Zanidatamab
Zanidatamab is an investigational HER2-targeted bispecific antibody
that can simultaneously bind two non-overlapping epitopes of the
HER2 receptor, known as biparatopic binding. This unique design and
increased binding results in multiple mechanisms of action,
including dual HER2 signal blockade, removal of HER2 protein from
the cell surface, and immune-mediated cytotoxicity leading to
encouraging antitumor activity in patients. Zanidatamab is
being developed in multiple clinical trials as a targeted treatment
option for patients with solid tumors that express
HER2. Zanidatamab is being developed by Jazz and
BeiGene, Ltd. (BeiGene) under license agreements from Zymeworks,
which first developed the molecule.
The U.S. Food and Drug Administration (FDA) has
granted Breakthrough Therapy designation for zanidatamab
development in patients with previously treated HER2 gene-amplified
biliary tract cancers (BTC), and two Fast Track designations for
zanidatamab: one as a single agent for refractory BTC and one in
combination with standard of care chemotherapy for 1L
gastroesophageal adenocarcinoma (GEA). Additionally,
zanidatamab has received Orphan Drug designations from FDA for the
treatment of BTC and GEA, as well as Orphan Drug designation from
the European Medicines Agency for the treatment of BTC
and gastric cancer. Zanidatamab was also granted Breakthrough
Therapy designation from the Center for Drug
Evaluation (CDE) in China.
About Biliary Tract Cancer
BTC, including gallbladder cancer and intrahepatic and extrahepatic
cholangiocarcinoma, account for <1% of all adult cancers
globally and are often associated with a poor
prognosis1,2. The human epidermal growth factor receptor
2 (HER2) is a well-validated target for antitumor therapy in other
cancers. Across the U.S., Europe,
and Japan, approximately 12,000
people are diagnosed with HER2+ BTC annually3,4,5,6.
About Jazz Pharmaceuticals
Jazz Pharmaceuticals plc
(Nasdaq: JAZZ) is a global biopharma company whose purpose is to
innovate to transform the lives of patients and their families. We
are dedicated to developing life-changing medicines for people with
serious diseases—often with limited or no therapeutic options. We
have a diverse portfolio of marketed medicines, including leading
therapies for sleep disorders and epilepsy, and a growing portfolio
of cancer treatments. Our patient-focused and science-driven
approach powers pioneering research and development advancements
across our robust pipeline of innovative therapeutics in oncology
and neuroscience. Jazz is headquartered in Dublin, Ireland with research and development
laboratories, manufacturing facilities and employees in multiple
countries committed to serving patients worldwide. Please visit
www.jazzpharmaceuticals.com for more information.
Jazz Pharmaceuticals plc Caution Concerning
Forward-Looking Statements
This press release contains
forward-looking statements, including, but not limited to,
statements related to our opportunity to deliver this treatment
option to the BTC community with the potential to be the first
HER2-targeted treatment specifically indicated for HER2+ BTC
and other statements that are not historical facts. These
forward-looking statements are based on Jazz Pharmaceuticals'
current plans, objectives, estimates, expectations and intentions
and inherently involve significant risks and uncertainties. Actual
results and the timing of events could differ materially from those
anticipated in such forward-looking statements as a result of these
risks and uncertainties, which include, without limitation, risks
and uncertainties associated with pharmaceutical product
development, and other risks and uncertainties affecting Jazz
Pharmaceuticals and its development programs, including those
described from time to time under the caption "Risk Factors" and
elsewhere in Jazz Pharmaceuticals plc's Securities and Exchange
Commission filings and reports (Commission File No. 001-33500),
including Jazz Pharmaceuticals' Annual Report on Form 10-K for the
year ended December 31, 2023, as
supplemented by our Quarterly Report on Form 10-Q for the quarter
ended March 31, 2024, and future
filings and reports by Jazz Pharmaceuticals. Other risks and
uncertainties of which Jazz Pharmaceuticals is not currently aware
may also affect Jazz Pharmaceuticals' forward-looking statements
and may cause actual results and the timing of events to differ
materially from those anticipated. The forward-looking statements
herein are made only as of the date hereof or as of the dates
indicated in the forward-looking statements, even if they are
subsequently made available by Jazz Pharmaceuticals on its website
or otherwise. Jazz Pharmaceuticals undertakes no obligation to
update or supplement any forward-looking statements to reflect
actual results, new information, future events, changes in its
expectations or other circumstances that exist after the date as of
which the forward-looking statements were made.
Contacts:
Jazz Media Contact:
Kristin
Bhavnani
Head of Global Strategic Brand Engagement
Jazz Pharmaceuticals plc
CorporateAffairsMediaInfo@jazzpharma.com
Ireland +353 1 637 2141
U.S. +1 215 867 4948
Jazz Investor Contact:
Andrea
N. Flynn, Ph.D.
Vice President, Head, Investor Relations
Jazz Pharmaceuticals plc
investorinfo@jazzpharma.com
Ireland +353 1 634 3211
U.S. +1 650 496 2717
References:
1 Valle JW, et al. Lancet 2021; 397:428-44
2 Siegel RL, et al. CA Cancer J Clin 2022;
72;7-33
3 BTC overall diagnosed patients as per SEER 22
4 Assumes anatomic subsites intrahepatic CCA,
extrahepatic CCA, gallbladder cancer, and BTC unspecified
5 Assumes HER2 positivity rates per
anatomical subsite from: Galdy, S., Lamarca, A., McNamara,
M.G. et al. Cancer Metastasis Rev 36, 141–157 (2017), Nobuyoshi Hiraoka, et al. Human Pathology,
Volume 105, 2020, Pages 9-19
6 Major markets: U.K, France, Germany, Spain, Italy.
Note: HER2+ BTC patients in Jazz-controlled commercial territories,
which includes Japan, and excludes
other certain Asia Pacific
countries licensed to BeiGene, Ltd
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SOURCE Jazz Pharmaceuticals plc