New England Journal of Medicine publishes positive results of the pivotal trial of Cablivi® (caplacizumab) for rare blood cl...
09 Enero 2019 - 4:00PM
New England Journal of Medicine publishes positive results of the pivotal trial of
Cablivi®
(caplacizumab) for rare blood clotting disorder
-
HERCULES Phase 3 trial met its primary endpoint,
demonstrating that treatment with Cablivi resulted in a
significantly faster time to platelet count response in patients
with acquired thrombotic thrombocytopenic purpura (aTTP) when
administered in conjunction with plasma exchange and
immunosuppression
-
Cablivi was associated with a significant
reduction in aTTP-related death, recurrence, or at least one major
thromboembolic event while patients were on treatment, the study's
first key secondary endpoint
-
Cablivi was approved by the European Commission
in August 2018
PARIS - January 9, 2019 - The
New England Journal of Medicine (NEJM) today published positive
results of the Phase 3 trial of Cablivi®
(caplacizumab) in adults with acquired thrombotic thrombocytopenic
purpura (aTTP).
aTTP is a rare, life-threatening autoimmune blood disorder
characterized by extensive clot formation in small blood vessels
throughout the body, leading to severe thrombocytopenia (very low
platelet count), microangiopathic hemolytic anemia (loss of red
blood cells through destruction), ischemia (restricted blood supply
to parts of the body) and widespread organ damage, especially in
the brain and heart.
The current treatment for aTTP consists of daily plasma exchange,
in which a patient's blood plasma is removed and replaced with
donor plasma, and immunosuppression. Even with currently available
treatments, patients continue to be at risk of developing acute
blood clotting conditions, such as stroke and heart attack, as well
as recurrence of disease.
Key findings of the HERCULES Phase 3 study of Cablivi
include:
-
On the primary endpoint, Cablivi significantly
reduced the time to platelet count normalization (p=0.01). At any
given time point during the study, patients receiving Cablivi were
1.55 times more likely to achieve normal platelet counts than
patients on placebo.
-
Treatment with Cablivi was associated with a 74
percent reduction in aTTP-related death, recurrence of aTTP, or at
least one major thromboembolic event compared with placebo
(p<0.001).
-
During the overall study period, patients
receiving Cablivi experienced a significantly lower number of aTTP
recurrences (67 percent reduction) compared to placebo
(p<0.001).
-
Refractory disease developed in 0 patients in
the Cablivi group versus 3 patients in the placebo group, although
this did not reach statistical significance (p=0.06).
-
Normalization of three organ-damage markers
(lactate dehydrogenase, cardiac troponin I, and serum creatinine)
occurred sooner in patients who received caplacizumab versus
placebo (p not tested due to hierarchical statistical
testing).
-
Results showed a clinically meaningful reduction
in the use of plasma exchange in patients treated with Cablivi
(average 5.8 days; 38 percent reduction) versus placebo (9.4 days),
as well as a shorter stay in the intensive care unit (65 percent
reduction) and hospital (31 percent reduction).
Cablivi demonstrated a safety profile consistent with what has been
previously reported and in line with its mechanism of action; this
included an increased risk of bleeding. The most frequently
reported bleeding-related adverse events were epistaxis and
gingival bleeding.
"aTTP
is a life-threatening disease, and the current treatment options do
not fully halt the extensive clot formation in small blood vessels
throughout the body, leaving patients at risk for significant
morbidity and early death," said Marie Scully, M.D, professor
of hematology at University College London Hospitals, and lead
author of the HERCULES study. "These results
demonstrate that Cablivi has the potential to address a major unmet
medical need and to help those facing the potentially devastating
consequences of this disorder."
HERCULES is a Phase 3 randomized, double-blind, placebo-controlled
study of Cablivi in patients with aTTP. In the study, 145 patients
were randomly assigned to Cablivi or placebo in conjunction with
plasma exchange and immunosuppression.
About Cablivi®
(caplacizumab)
Cablivi is an anti-vWF Nanobody, which inhibits the interaction
between ultra-large von Willebrand Factor (vWF) multimers and
platelets and, therefore, stops the formation and accumulation of
the micro-clots that cause the thrombocytopenia, tissue ischemia,
and organ dysfunction in aTTP. Cablivi was developed by Ablynx, a
Sanofi company.
Cablivi was approved by the European Commission in August 2018 for
the treatment of adults experiencing an episode of aTTP. It is the
first therapeutic specifically indicated for the treatment of
aTTP.
Additionally, the U.S. Food and Drug Administration (FDA) has
accepted for priority review the Biologics License Application for
Cablivi for treatment of patients 18 years of age and older
experiencing an episode of aTTP. The target action date for the FDA
decision is February 6, 2019.
|
About Sanofi
Sanofi is dedicated to supporting people through their health
challenges. We are a global biopharmaceutical company focused on
human health. We prevent illness with vaccines, provide innovative
treatments to fight pain and ease suffering. We stand by the few
who suffer from rare diseases and the millions with long-term
chronic conditions.
With more than 100,000 people in 100 countries, Sanofi is
transforming scientific innovation into healthcare solutions around
the globe.
Sanofi, Empowering Life
|
Media Relations Contact Ashleigh
Koss
Tel.: +1 (908) 981-8745
Ashleigh.Koss@sanofi.com
|
Investor Relations Contact George
Grofik
Tel.: +33 (0)1 53 77 45 45
ir@sanofi.com
|
Sanofi Forward-Looking Statements
This press release contains forward-looking
statements as defined in the Private Securities Litigation Reform
Act of 1995, as amended. Forward-looking statements are statements
that are not historical facts. These statements include projections
and estimates regarding the marketing and other potential of the
product, or regarding potential future revenues from the product.
Forward-looking statements are generally identified by the words
"expects", "anticipates", "believes", "intends", "estimates",
"plans" and similar expressions. Although Sanofi's management
believes that the expectations reflected in such forward-looking
statements are reasonable, investors are cautioned that
forward-looking information and statements are subject to various
risks and uncertainties, many of which are difficult to predict and
generally beyond the control of Sanofi, that could cause actual
results and developments to differ materially from those expressed
in, or implied or projected by, the forward-looking information and
statements. These risks and uncertainties include among other
things, unexpected regulatory actions or delays, or government
regulation generally, that could affect the availability or
commercial potential of the product, the absence of guarantee that
the product will be commercially successful, the uncertainties
inherent in research and development, including future clinical
data and analysis of existing clinical data relating to the
product, including post marketing, unexpected safety, quality or
manufacturing issues, competition in general, risks associated with
intellectual property and any related future litigation and the
ultimate outcome of such litigation, and volatile economic
conditions, as well as those risks discussed or identified in the
public filings with the SEC and the AMF made by Sanofi, including
those listed under "Risk Factors" and "Cautionary Statement
Regarding Forward-Looking Statements" in Sanofi's annual report on
Form 20-F for the year ended December 31, 2017. Other than as
required by applicable law, Sanofi does not undertake any
obligation to update or revise any forward-looking information or
statements.
|
Press release (PDF)
This
announcement is distributed by West Corporation on behalf of West
Corporation clients.
The issuer of this announcement warrants that they are solely
responsible for the content, accuracy and originality of the
information contained therein.
Source: Sanofi via Globenewswire
Sanofi (EU:SAN)
Gráfica de Acción Histórica
De Mar 2024 a Abr 2024
Sanofi (EU:SAN)
Gráfica de Acción Histórica
De Abr 2023 a Abr 2024