DUBLIN, Oct. 3, 2016 /PRNewswire/ -- Jazz Pharmaceuticals
plc (Nasdaq; JAZZ) today announced the initiation of a rolling
submission of a New Drug Application (NDA) to the United States (U.S.) Food and Drug
Administration (FDA) on September 30,
2016, seeking marketing approval of Vyxeos™ (cytarabine and
daunorubicin liposome injection), an investigational agent for the
treatment of acute myeloid leukemia (AML). The company
expects to complete the submission of the NDA by early 2017, and
will request a priority review.
"Our initiation of the rolling NDA submission for Vyxeos brings
us closer to an important goal of providing a new treatment option
for patients with acute myeloid leukemia, a devastating and
life-threatening disease," said Karen Smith
M.D., Ph.D., global head of research and development and
chief medical officer at Jazz Pharmaceuticals. "AML
represents a disease area within the hematological cancers, where
there remains a significant unmet medical need. Vyxeos, if
approved, would be the first new treatment demonstrating a
significant advancement in treating AML in over 20 years."
Vyxeos was granted Breakthrough Therapy Designation in
May 2016 for the treatment of adults
with therapy-related AML or AML with myelodysplasia-related
changes. The FDA grants Breakthrough Therapy designation to
expedite the development and review of new medicines that are
intended to treat serious or life-threatening diseases when the
clinical evidence indicates that the drug may demonstrate
substantial improvement over existing therapies on at least one
clinically significant endpoint. The Breakthrough Therapy
designation allows a company to submit individual sections of its
NDA for review by the FDA as they are completed rather than waiting
until the entire application is complete before it can be submitted
and reviewed.
Celator Pharmaceuticals, Inc. completed and announced the
results of its Phase 3 trial evaluating Vyxeos in patients with
high-risk AML in March 2016.
Jazz Pharmaceuticals completed the acquisition of Celator
Pharmaceuticals in July 2016.
About Acute Myeloid Leukemia
Acute Myeloid Leukemia (AML) is a rapidly progressing and
life-threatening blood cancer that rises in frequency with
age.1 The American Cancer Society estimates that
there will be 19,950 new cases of AML and 10,430 deaths from AML in
the U.S. in 2016.2 In the European Union, the
number of new cases is estimated to be 20,100 in
2016.3
The median age at diagnosis is 67 and with rising age there is
progressive worsening of prognosis.1,4 Advancing
age is associated with increasing risk of specific
chromosomal/mutational changes and risk of pre-malignant marrow
disorders which give rise to more aggressive and less responsive
forms of AML.5,6 As patients age there is also
reduced tolerance for intensive chemotherapy.7 As
a consequence, advances in supportive care, intensive chemotherapy,
and bone marrow transplantation have primarily benefitted younger
patients with approximately one third of patients 18-60 years of
age achieving cure.5,7 Older patients have not
achieved higher rates of cure or improved upon a 5-year survival
rate of 10-20% in spite of 40 years of research. 7,
8
About Vyxeos
Vyxeos™ (cytarabine and daunorubicin liposome injection), or
CPX-351, is a combination of cytarabine and daunorubicin
encapsulated within a nano-scale liposome at a 5:1 molar
ratio. Vyxeos was granted orphan drug status by the FDA and
the European Commission for the treatment of acute myeloid
leukemia. Vyxeos was granted Breakthrough Therapy Designation
for the treatment of adults with therapy-related AML or AML with
myelodysplasia-related changes and was also granted Fast Track
designation by the FDA for the treatment of older patients with
secondary AML.
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) and Defitelio®
(defibrotide sodium) in the U.S. and markets Erwinase® and
Defitelio® (defibrotide) in countries outside the U.S. 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 Jazz
Pharmaceuticals' plans to complete a rolling NDA submission for
Vyxeos in the U.S. and the timing thereof, 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, the
inherent uncertainty associated with the regulatory approval
process, including the risk that the company may be unable to
obtain or maintain regulatory approval for Vyxeos in the U.S., and
other risks and uncertainties affecting the company, 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, 2016 and
future filings and reports by the company. Other risks and
uncertainties of which Jazz Pharmaceuticals is not currently aware
may also affect its forward-looking statements and may cause actual
results and 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 does not undertake any
obligation to update or supplement any forward-looking statements
to reflect actual results, new information, future events, changes
in expectations or other circumstances that exist after the date as
of which the forward-looking statements were made.
References:
1 American Cancer Society: Key Statistics
About Acute Myeloid Leukemia; information pulled on August 18, 2016 from
http://www.cancer.org/cancer/leukemia-acutemyeloidaml/detailedguide/leukemia-acute-myeloid-myelogenous-key-statistics
2 SEER Stat Fact Sheets - AML. (2016)
http://seer.cancer.gov/statfacts/html/amyl.html. Accessed
9/19/16.
3 Decision Resources Group AML Disease
Report. 2015 November.
4 Baer MR, et al. Leukemia, 2011 May;
25(5):10.1038/eu.2011.9.
5 Ferrara F, et al., Lancet, 2013
Feb 9 – 15 (381):484-495.
6 Dohner H, et al. Blood, 2010;
115(3):453-474.
7 Stone RM, et al., Hematology Am Soc Hematol
Educ Program, 2004:98-117.
8 Kadia TM, Ann Oncol. 2016 May 27 (5),
770-8.
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