DUBLIN, Sept. 30, 2015 /PRNewswire/ -- Jazz
Pharmaceuticals plc (Nasdaq: JAZZ) today announced that
the United States (U.S.) Food and
Drug Administration (FDA) has accepted for filing with Priority
Review its recently submitted New Drug Application (NDA) for
defibrotide. Defibrotide is an investigational agent proposed
for the treatment of patients with hepatic veno-occlusive disease
(VOD), also known as sinusoidal obstruction syndrome (SOS), with
evidence of multi-organ dysfunction (MOD) following hematopoietic
stem-cell transplantation (HSCT).
Priority Review status is designated for drugs that may offer
major advances in treatment or provide a treatment where no
adequate therapy exists. Based on timelines established by
the Prescription Drug User Fee Act (PDUFA), FDA review of the NDA
is expected to be completed by March
31, 2016.
"The FDA's acceptance for filing and Priority Review status of
the NDA for defibrotide is an important milestone for Jazz and
reflects our commitment to bringing meaningful medicines to
patients who have significant unmet needs," said Karen Smith,
M.D., Ph.D., Global Head of Research and Development and Chief
Medical Officer of Jazz Pharmaceuticals. "We look forward to
continuing to work closely with the FDA to obtain approval for
defibrotide for patients with hepatic VOD with evidence of MOD in
the U.S. as quickly as possible, as there are no other approved
therapies for treating this rare, often fatal complication of
HSCT."
The NDA includes safety and efficacy data from three clinical
studies of defibrotide for the treatment of hepatic VOD with MOD
following HSCT, as well as a retrospective review of registry data
from the Center for International Blood and Marrow Transplant
Research. The safety database includes over 900 patients
exposed to defibrotide in the clinical development program for the
treatment of hepatic VOD.
"We applaud the FDA for working with Jazz in accepting this
application for a timely review as bone marrow transplant patients
who develop VOD with MOD currently have no effective options for
this potentially life-threatening syndrome," said Susan K. Stewart, Executive Director, BMT
InfoNet (Blood & Marrow Transplant Information Network).
About VOD
HSCT is a potentially curative procedure to treat patients with
malignant and non-cancerous hematologic disorders such as leukemia,
lymphoma and aplastic anemia, congenital immunodeficiencies and
metabolic disorders.1 Hepatic VOD is a rare, early
and life-threatening complication of HSCT. VOD in association with
MOD has a mortality rate that exceeds 80%.2
About Defibrotide
In the U.S., defibrotide is an investigational drug for the
treatment of patients with hepatic VOD with evidence of MOD
following HSCT. Defibrotide was granted Orphan Drug
Designation by the FDA in May 2003
and has Fast Track designation. Defibrotide is being made
available as an investigational new drug (IND) free of charge
through an expanded access Treatment Protocol.
The ongoing expanded access Treatment Protocol is currently
enrolling patients diagnosed with VOD in the U.S. Expanded
access programs are part of an effort by the FDA and the
pharmaceutical industry to make investigational drugs available for
the treatment of serious or life threatening diseases in people
with limited treatment options. For information about the
defibrotide study, contact Erin
Tokunaga at erin.tokunaga@jazzpharma.com or Lam Calderon
(1.312.706.6240; 0265-002Gentium@iconplc.com); or visit
www.clinicaltrials.gov (Identifier: NCT00628498).
In Europe, defibrotide is
marketed under the name Defitelio®▼(defibrotide).
In October 2013, the European Commission granted
marketing authorization to Defitelio under exceptional
circumstances for the treatment of severe hepatic VOD in patients
undergoing HSCT therapy. It is the first and only approved
treatment in Europe for severe
hepatic VOD. In Europe, Defitelio is indicated in
patients over one month of age. It is not indicated in
patients with hypersensitivity to defibrotide or any of its
excipients or with concomitant use of thrombolytic therapy.
Please consult the Defitelio Summary of Product Characteristics
(SmPC) before prescribing, particularly in relation to use of
medicinal products that increase the risk of hemorrhage,
concomitant systemic anticoagulant therapy, medicinal products that
affect platelet aggregation, use in patients who have or develop
clinically significant acute bleeding requiring blood transfusion,
and patients who have hemodynamic instability.
The most frequent adverse events observed during pre-marketing
use were hemorrhage, hypotension and coagulopathy. Please
consult the Defitelio SmPC for the full list of all side
effects reported with Defitelio.
▼This medicinal product is subject to additional
monitoring. This will allow quick identification of new
safety information. Healthcare professionals are asked to
report any suspected adverse reactions via the national reporting
system found under section 4.8 of the SmPC.
(http://www.ema.europa.eu/ema/index.jsp?curl=/pages/medicines/human/medicines/002393/human_med_001646.jsp)
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 and
Erwinaze® (asparaginase Erwinia chrysanthemi) 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 the expected
timing of and efforts in connection with obtaining FDA approval of
Jazz Pharmaceuticals' NDA for defibrotide 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, among others, risks and uncertainties
associated with the difficulty and uncertainty of pharmaceutical
product development, including the timing thereof, and the
uncertainty of clinical success; the inherent uncertainty
associated with the regulatory approval process, including the risk
that the company may be unable to obtain regulatory approval for
defibrotide in the U.S. in a timely manner or at all; 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 most recent Annual Report on Form 10-K or
Quarterly Report on Form 10-Q 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 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:
1Gratwohl A et al. JAMA 2010;
303:1617-1624 (Ikehara, 2002).
2Coppell et al. 2010, Carreras et al. 2011.
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