Jazz
Pharmaceuticals plc 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.
Market News and Data brought to you by Benzinga APIs© 2024 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.
Comments
Loading...
Benzinga simplifies the market for smarter investing
Trade confidently with insights and alerts from analyst ratings, free reports and breaking news that affects the stocks you care about.
Join Now: Free!
Already a member?Sign in