Vyxeos granted accelerated assessment review status from
European Medicines Agency's Committee for Medicinal Products for
Human Use
DUBLIN, Nov. 3, 2017 /PRNewswire/ -- Jazz Pharmaceuticals
plc (Nasdaq: JAZZ) today announced the submission of a Marketing
Authorization Application (MAA) to the European Medicines Agency's
Committee for Medicinal Products for Human Use (CHMP) for Vyxeos™
(daunorubicin and cytarabine) powder for concentrate for infusion
to treat adults with high-risk acute myeloid leukemia (AML) defined
as therapy-related AML (t-AML) or AML with myelodysplasia-related
changes (AML-MRC).
The CHMP granted Vyxeos accelerated assessment, which is
designed to reduce the review timeline for products of major
interest for public health and therapeutic innovation.
Vyxeos also received Promising Innovative Medicine (PIM)
designation from the Medicines and Healthcare Products Regulatory
Agency in the United Kingdom. A
PIM designation is an early indication that a medicinal product is
a promising candidate for the Early Access to Medicines Scheme
(EAMS), intended for the treatment, diagnosis or prevention of a
life-threatening or seriously debilitating condition with the
potential to address an unmet medical need.
"If approved, Vyxeos will become the first
new chemotherapy treatment option specifically for European
patients with therapy-related AML or AML with
myelodysplasia-related changes," said Karen Smith, M.D., Ph.D., executive vice
president, research and development and chief medical officer
at Jazz Pharmaceuticals. "We are passionate about
bringing a new treatment option for high-risk AML to the
appropriate patients in the EU as quickly as possible and look
forward to working with the CHMP during this review process."
The MAA for Vyxeos includes clinical data from five
studies, including the pivotal Phase 3 study. Data from the Phase 3
study, which demonstrated a statistically significant improvement
in overall survival for Vyxeos versus standard of care, were
presented at the American Society of Clinical Oncology Annual
Meeting in June 2016.
"Despite numerous advances in the treatment of cancer in
Europe, patients with high-risk
AML defined as t-AML or AML-MRC have limited treatment options and
some of the lowest survival rates compared to people with other
forms of AML," said Dr. Nigel
Russell, Professor of Haematology, Faculty of Medicine &
Health Sciences at the University of Nottingham. "The hematology oncology community
has worked tirelessly to better understand the causes and disease
pathways associated with AML and we look forward to the arrival of
innovative new treatment options."
About Vyxeos™
Vyxeos daunorubicin 2.2 mg/mL and cytarabine 5 mg/mL powder
for concentrate for infusion is an advanced liposomal formulation
that delivers a fixed-ratio (1:5) of daunorubicin and cytarabine to
the bone marrow that has been shown to have synergistic effects at
killing leukemia cells in vitro and in animal models. Vyxeos
is the first product developed with the company's proprietary
CombiPlex® platform, which enables the design and rapid
evaluation of various combinations of therapies. Vyxeos
received Orphan Drug Designation by the European Commission in
January 2012 and by the U.S. Food and
Drug Administration (FDA) in September
2008 for the treatment of AML. Vyxeos received U.S.
FDA approval on August 3, 2017 for
the treatment of adults with newly-diagnosed t-AML or
AML-MRC.
About AML
Acute myeloid leukemia (AML) is a blood
cancer that begins in the bone marrow, which produces most of the
body's new blood cells.1 AML cells crowd out healthy
cells and move aggressively into the bloodstream to spread cancer
to other parts of the body.2 The median age at diagnosis
is 68 years old, with rising age associated with a progressively
worsening prognosis.3-4 There is also a reduced
tolerance for intensive chemotherapy as patients age.5
Patients with t-AML or AML-MRC have few treatment options and some
of the lowest survival rates compared to people with other forms of
leukemia.6-7 A hematopoietic stem cell transplant (HSCT)
may be a curative treatment option for patients.8
About Jazz Pharmaceuticals plc
Jazz Pharmaceuticals
plc (Nasdaq: JAZZ) is an international biopharmaceutical company
focused on improving patients' lives by identifying, developing and
commercializing meaningful products that address unmet medical
needs. The company has a diverse portfolio of products and
product candidates with a focus in the areas of sleep and
hematology/oncology. In these areas, Jazz Pharmaceuticals
markets Xyrem® (sodium oxybate) oral solution,
Erwinaze® (asparaginase Erwinia chrysanthemi),
Defitelio® (defibrotide sodium) and Vyxeos™
(daunorubicin and cytarabine) liposome for injection in the U.S.
and markets Erwinase® and Defitelio®
(defibrotide) in countries outside the U.S. For country-specific
product labels, please visit www.jazzpharma.com/products. For more
information, please visit www.jazzpharmaceuticals.com.
"Safe Harbor" Statement under the Private Securities
Litigation Reform Act of 1995
This press release contains
forward-looking statements, including, but not limited to,
statements related to Vyxeos as a treatment option for European
patients with t-AML or AML-MRC, the expected impact of accelerated
assessment review status on the Vyxeos MAA, and other statements
that are not historical facts. These forward-looking statements are
based on the company's 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: the regulatory approval process,
including the company's ability to maintain accelerated assessment
review status for the Vyxeos MAA or obtain approval for Vyxeos in
the EU in a timely manner or at all; and the manufacture and
effective commercialization of Vyxeos in the EU; and other risks
and uncertainties affecting the company 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 the company's Quarterly Report on
Form 10-Q for the quarter ended June 30,
2017 and future filings and reports by the company. Other
risks and uncertainties of which the company is not currently aware
may also affect the company's 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 the company on its website or otherwise. The company
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.
References:
1. National Cancer Institute. General
Information About Adult Acute Myeloid Leukemia
https://www.cancer.gov/types/leukemia/patient/adult-aml-treatment-pdq
Accessed June 7, 2017.
2. American Cancer Society. What is Acute Myeloid Leukemia?
https://www.cancer.org/cancer/acute-myeloid-leukemia/about/what-is-aml.html
Accessed March 20, 2017.
3. SEER Stat Facts: AML. 2017.
4. Baer MR, et al., Leukemia, 2011 May;
25(5):10.1038/eu.2011.9.
5. Klepin HD. Hematology Am Soc Hematol Educ Program. 2014
(1):8-13.
6. Pinto AC, et al. Current Cancer Treatment - Novel Beyond
Conventional Approaches. 2011; 693-714.
7. Smith SM, et al. Blood. 2003; 102(1):43-52.
8. Peccatori, J and Ciceri, F. Haematologica. 2010 Jun; 95(6):
857–859. doi: 10.3324/haematol.2010.023184.
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