-- Expects to complete submission in the first half of
2015
DUBLIN, Dec. 11, 2014 /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) for defibrotide for the treatment of
severe hepatic veno-occlusive disease (VOD) in patients undergoing
hematopoietic stem-cell transplantation (HSCT) therapy.
Defibrotide has been granted Fast Track Designation to treat severe
VOD by the FDA.
"Our start of the NDA submission for defibrotide marks an
important step forward in our efforts to provide a treatment option
for patients in the U.S. who develop this rare, life-threatening
complication of HSCT," said Jeffrey
Tobias, M.D., executive vice president and chief medical
officer of Jazz Pharmaceuticals. "We expect to complete the
submission of the NDA in the first half of 2015, at which time we
will be requesting a Priority Review of the application, and we
will continue to work closely with the FDA as we seek approval of
the NDA. As part of our commitment to ensure that eligible
patients have access to defibrotide as we pursue U.S. approval, we
will continue to provide patients access to defibrotide through an
expanded access treatment protocol that is open under an ongoing
investigational new drug application in the U.S."
The Fast Track Designation is designed to facilitate the
development and expedite the review of drugs that treat serious,
life-threatening conditions and that address unmet medical
needs. The Fast Track process 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.
Earlier this year, Jazz Pharmaceuticals acquired the rights to
defibrotide in the U.S. and other markets in North America, South
America and Central America. Jazz Pharmaceuticals
markets defibrotide in Europe
under the name Defitelio®▼ (defibrotide). Defitelio is the
first and only licensed product in Europe for the treatment of severe hepatic VOD
in patients over one month of age undergoing HSCT therapy.
About VOD
Veno-occlusive disease (VOD) is an early complication in
patients undergoing HSCT therapy. In its severe form, VOD can
be life-threatening and is associated with multi-organ failure; it
is fatal in over 80% of patients.1,2 HSCTs are
performed with curative intent in patients with hematological
malignancies, selected solid tumors and some non-malignant
disorders, such as serious
hemoglobinopathies.3,4 In the U.S.,
defibrotide was granted Orphan Drug Designation to treat and
prevent VOD by the FDA in May
2003.
About Defibrotide
In October 2013,
the European Commission granted marketing authorization
under exceptional circumstances for Defitelio®▼ (defibrotide) for
the treatment of severe hepatic VOD in patients undergoing HSCT
therapy. It is indicated in patients over one month of age.
Defitelio 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)
[http://www.ema.europa.eu/ema/index.jsp?curl=/pages/medicines/human/medicines/002393/human_med_001646.jsp] 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.5 Please also 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.
About Jazz Pharmaceuticals plc
Jazz Pharmaceuticals plc (Nasdaq: JAZZ) is a specialty
biopharmaceutical company focused on improving patients' lives by
identifying, developing and commercializing differentiated products
that address unmet medical needs. The company has a diverse
portfolio of products and/or product candidates in the areas of
sleep, hematology/oncology, pain and psychiatry. The
company's U.S. marketed products in these areas include: Xyrem®
(sodium oxybate) oral solution, Erwinaze® (asparaginase Erwinia
chrysanthemi), Prialt® (ziconotide) intrathecal infusion,
Versacloz® (clozapine) oral suspension, FazaClo® (clozapine, USP)
HD and FazaClo LD. Jazz Pharmaceuticals also has a number of
products marketed outside the United
States, including Erwinase® and Defitelio® (defibrotide).
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
defibrotide 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 expectations 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 risks that the
company may be required to conduct additional time-consuming and
costly clinical trials prior to submission of the NDA or as a
condition of regulatory approval of defibrotide in the U.S. and
that the company may otherwise be unable to obtain or maintain any
regulatory approval for defibrotide in the U.S., and those other
risks detailed 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 Quarterly Report on Form 10-Q
for the quarter ended September 30,
2014 and future filings and reports by the company.
Jazz Pharmaceuticals undertakes no duty or obligation to update any
forward-looking statements contained in this press release as a
result of new information, future events or changes in its
expectations.
References
1. Carreras E. EBMT-ESH
Handbook 2012. Chapter 11: Early complications after HSCT.
2. Coppell JA, et al. Biol Blood Marrow Transplant.
2010;16:157-168.
3. Tsakiris DA, Tichelli A. Best Pract Res Clin
Haematol. 2009;22:137-145.
4. Majhail NS, et al. Bone Marrow Transplant.
2013;48:294-300.
5. Defitelio® Summary of product characteristics,
2013.