DUBLIN and VILLA GUARDIA,
Italy, March 31, 2014 /PRNewswire/ -- Jazz
Pharmaceuticals plc (Nasdaq: JAZZ) and Gentium S.p.A., a Jazz
Pharmaceuticals company, today announced the commencement of the
European commercial launch of Defitelio®▼
(defibrotide), the first licensed product for the treatment of
severe hepatic veno-occlusive disease (severe VOD or sVOD) in
patients over one month of age undergoing haematopoietic stem cell
transplantation (HSCT)
therapy.1 The companies have
launched Defitelio in Germany and
Austria and expect to
continue the launch in 27 additional European countries on a
rolling basis during 2014 and 2015.
Severe VOD, one of the most serious early complications in HSCT
therapy, is associated with multi-organ failure and is fatal in
over 80% of
patients.2,3 HSCTs
are performed with curative intent in patients with haematological
malignancies, selected solid tumours and some non-malignant
disorders, such as serious haemoglobinopathies.4,5
"The commercial availability of Defitelio as the first medicine
licensed for the treatment of sVOD in Europe is an important step forward for
patients with this life-threatening condition," said Bruce C. Cozadd, chairman and CEO at Jazz
Pharmaceuticals plc. "Additionally, this European launch
represents a key milestone for the combined Jazz Pharmaceuticals
and Gentium team following the acquisition of Gentium by Jazz
Pharmaceuticals earlier this year, and reinforces our commitment to
bringing important therapies to patients who have significant unmet
medical needs in the areas of haematology and oncology."
"Severe VOD is a complex and unpredictable disease, and its
impact on patients, physicians and resources is substantial.
Early and effective intervention is crucial in saving lives
and limiting the potentially significant burden of this disease,
and physicians have been eagerly awaiting the commercial
availability of Defitelio in Europe," said Professor Mohamad Mohty, President-Elect of the EBMT and
Professor of Haematology, Saint-Antoine Hospital and University
Pierre & Marie Curie,
Paris.
The efficacy of Defitelio to treat sVOD in HSCT patients is
supported by data from a pivotal, multi-centre Phase 3 trial that
evaluated Defitelio for the treatment of sVOD compared with a
historical control group of patients who had received standard
supportive care.1 In this trial, Defitelio was
shown to provide a significant increase in survival rates for
patients with sVOD in HSCT. The results demonstrated a 52%
increase in survival at 100 days after transplantation for patients
treated with Defitelio compared to patients in the historical
control group (38.2% in the Defitelio group vs. 25.0% in the
historical control group; p=0.0341).1
In the clinical trial, 23.5% of patients treated
with Defitelio achieved complete response at 100 days after
transplantation versus 9.4% of patients in the historical control
group (p=0.013).1
The efficacy data from this pivotal trial are supported with
data from a Phase 2 dose-finding study, as well as data from the
International Compassionate Use Programme and an interim analysis
(subset of patients with sVOD) of an ongoing, open-label treatment
investigational new drug (IND) study being conducted in
the United States
(U.S.).1 Additionally, data derived from an
independent registry in the U.S. supported the European approval of
Defitelio for use in patients with sVOD.1
Treatment with Defitelio has generally been well tolerated in
all age groups.1,6 In the Phase 3 pivotal
trial, the overall incidence of adverse events was similar in the
Defitelio treatment group and in the control
group.7 The most frequent adverse events
observed during pre-marketing use were haemorrhage, hypotension and
coagulopathy.1 Please consult the Defitelio SmPC
for the full list of all side effects reported with Defitelio.
▼ This medicinal product is subject to
additional monitoring.
About Defitelio®▼
(defibrotide)
In October
2013, the European Commission granted Marketing
Authorisation under exceptional circumstances for
Defitelio®▼ (defibrotide) for the
treatment of severe hepatic veno-occlusive disease in
haematopoietic stem-cell transplantation 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.
In addition to its existing approved indication in the European
Union (EU), defibrotide has the potential to be developed for
approval in countries outside the EU and in other indications.
Defibrotide has been granted orphan drug designation to treat and
prevent VOD by the U.S. Food and Drug Administration (FDA), by the
European Medicines Agency (EMA) and by the Korean Ministry of Food
and Drug Safety (MFDS), orphan drug designation for the treatment
of VOD by the Commonwealth of Australia-Department of Health, and
Fast Track designation to treat sVOD by the FDA. Sigma-Tau
Pharmaceuticals, Inc. has licensed the rights to commercialise
defibrotide for the treatment and prevention of VOD in North America, Central America and South America, subject to receipt of Marketing
Authorisation in the applicable territory.
Please consult the Defitelio SmPC before prescribing,
particularly in relation to use of medicinal products that increase
the risk of haemorrhage, 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
haemodynamic instability.
About VOD
Hepatic veno-occlusive disease (VOD) is an
early complication in patients undergoing HSCT. In its severe
form, VOD can be life-threatening and is associated with
multi-organ failure and is fatal in over 80% of patients.
2,3HSCTs are performed with curative intent in
patients with haematological malignancies, selected solid tumours
and some non-malignant disorders, such as serious
haemoglobinopathies.4,5 In the EU, VOD is
designated as a rare disease, affecting less than five in 10,000
people. Studies have reported a wide range of incidence rates
for VOD. Generally, data indicate that approximately 14% of
patients undergoing HSCT develop VOD.3
About Jazz Pharmaceuticals plc
Jazz Pharmaceuticals
plc (Nasdaq: JAZZ) is a specialty biopharmaceutical company focused
on improving patients' lives by identifying, developing and
commercialising differentiated products that address unmet medical
needs. The company has a diverse portfolio of products and/or
product candidates in the areas of sleep, haematology/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.
About Gentium S.p.A.
Gentium S.p.A., a majority owned
indirect subsidiary of Jazz Pharmaceuticals plc, developed
Defitelio®▼ (defibrotide) and markets
Defitelio in Europe. Gentium
is located in Villa Guardia (Como), Italy. For more information, please
visit www.gentium.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 therapeutic and commercial potential of
Defitelio®▼ (defibrotide), the
expected launch of Defitelio in additional European countries and
the timing thereof, the potential for defibrotide to be developed
for approval in countries outside the EU and in other indications
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, risks and uncertainties associated
with the company's ability to realise the anticipated revenues from
Defitelio; the company's ability to successfully launch and
commercialise Defitelio in a timely manner in additional European
countries, including possible delays and unforeseen difficulties in
obtaining pricing and reimbursement approvals for Defitelio in any
of these countries and the possibility that the approved level of
governmental pricing and reimbursement for Defitelio in any of
these countries may be lower than the company's estimates; the
company's ability to successfully manage the risks associated with
integrating Defitelio into the company's product portfolio; the
company's ability to obtain regulatory approval for defibrotide in
other countries, including the United
States, and in other indications; 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 Annual Report on Form 10-K for the year ending in
December 31, 2013 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.
SOURCE Jazz Pharmaceuticals plc and Gentium S.p.A.
▼ This medicinal product
is subject to additional monitoring.
References
1 Defitelio® Summary of product characteristics,
2013
2 Carreras E. Chapter 11: Early complications after
HSCT. EBMT-ESH Handbook 2012
3 Coppell JA et al. Biol Blood Marrow Transplant
2010;16:157–168
4 Tsakiris DA & Tichelli A. Best Pract Res Clin
Haematol 2009;22:137–145
5 Majhail NS et al. Bone Marrow Transplant
2013;48:294–300
6 Richardson PG et al. Expert Opin Drug Saf
2013;12:123–136
7 Richardson PG et al. Blood (ASH Annual Meeting
Abstracts) 2009;114:654.
SOURCE Jazz Pharmaceuticals plc; Gentium S.p.A.