Sanofi: FDA approves Cablivi® (caplacizumab-yhdp), the first Nanobody®-based medicine, for adults with acquired thrombotic ...
06 Febrero 2019 - 10:43AM
FDA approves
Cablivi®
(caplacizumab-yhdp), the first Nanobody®-based
medicine, for adults with acquired thrombotic thrombocytopenic
purpura (aTTP)
-
First medicine approved in the U.S. specifically
for the treatment of aTTP, a rare blood-clotting disorder
-
Cablivi is the first U.S. approval for Sanofi's
new rare blood disorders franchise
PARIS - February 6, 2019 - The U.S. Food and
Drug Administration (FDA) has approved Cablivi®
(caplacizumab-yhdp) in combination with plasma exchange and
immunosuppression for the treatment of acquired thrombotic
thrombocytopenic purpura (aTTP) in adults. Cablivi is the
first FDA approved therapy specifically indicated for the treatment
of aTTP.
"The U.S.
approval of Cablivi provides a much-needed treatment option for
people facing this challenging disease. There have been
limited medicines available to treat aTTP until now," says
Olivier Brandicourt, M.D., Chief Executive Officer, Sanofi. "Cablivi marks the first U.S. approval in our newly
formed rare blood disorders franchise, and we look forward to
continuing to provide important medicines for people living with
these very serious diseases."
Cablivi targets von Willebrand
factor (vWF), a protein in the blood involved in hemostasis.
It is designed to inhibit the interaction between vWF and
platelets. Cablivi is an anti-vWF Nanobody and Sanofi's first
Nanobody®-based
medicine to receive approval in the U.S.
Nanobodies® are
a novel class of proprietary therapeutic
proteins based on single-domain antibody fragments that
contain the unique structural and functional properties of
naturally-occurring heavy chain only antibodies.
Cablivi received FDA Fast Track
designation and was evaluated under Priority Review, which is
reserved for medicines that represent significant improvements in
safety or efficacy in treating serious conditions.
An Unmet Need in
a Rare Blood Disorder
aTTP is a rare, life-threatening, autoimmune blood disorder.
aTTP is considered an urgent, medical emergency. For some
patients, resuscitative measures might be required and the
immediate outcome might not be predictable. In most
cases, patients are routinely treated in intensive care units
during the first few days following their aTTP diagnosis. It is
estimated that up to 20% of patients die from TTP episodes, despite
currently available treatments (plasma exchange and
immunosuppression), with most deaths occurring within 30 days of
diagnosis. In the U.S., aTTP affects fewer than 2,000 adults
each year.
"aTTP is a very
severe, life-threatening disease. For those faced with this
rare diagnosis, the treatment and care can be difficult and the
threat of recurrence is ever-present," said Spero R. Cataland,
M.D., Professor of Internal Medicine, Division of Hematology,
Wexner Medical Center at the Ohio State University. "Cablivi provides new hope for adults in the U.S.
suffering with aTTP and provides a much needed treatment option to
help effectively manage aTTP episodes."
In aTTP, accumulation of
ultra-large vWF causes 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), and ischemia (restricted
blood supply to parts of the body).
Cablivi Clinical
Program and Results
The approval of Cablivi in the
U.S. is based on the results of the pivotal multicentre,
randomized, double-blind, placebo-controlled Phase 3 clinical study
known as HERCULES. This trial evaluated the efficacy of Cablivi in
combination with plasma exchange and immunosuppressive therapy
(n=72) versus placebo, plasma exchange and immunosuppressive
therapy (n=73) in 145 adults experiencing an episode of aTTP.
In the HERCULES study, treatment
with Cablivi in combination with plasma exchange and
immunosuppression resulted in a significantly shorter time to
platelet count response versus plasma exchange and
immunosuppression alone (Hazard Ratio 1.55 [1.10; 2.20] p=0.01),
the study's primary efficacy endpoint; in secondary endpoints,
Cablivi showed a significant reduction on a composite endpoint of
aTTP-related death, recurrence of aTTP, or a major thromboembolic
event during study drug treatment versus plasma exchange and
immunosuppression alone (12.7% vs. 49.3%; p <0.0001); and a
significantly lower percentage of aTTP recurrences in the overall
study period versus plasma exchange and immunosuppression alone
(13% vs. 38%; p<0.001). Results of this study were published in
the New England Journal of Medicine in January
2019.
In the HERCULES and TITAN (Phase
2) clinical trials, the most frequently reported adverse reactions
were epistaxis (bleeding from the nose) 29%, headache 21% and
gingival (gums) bleeding 16%. In the placebo group, two
deaths were reported in the TITAN study and three deaths in the
HERCULES study. No deaths were reported during the study drug
treatment period in the Cablivi group in the TITAN and HERCULES
studies. However, one death was reported in the HERCULES study
during the treatment free follow up period, which was determined
not to be Cablivi treatment related.
About
Cablivi
Cablivi should be administered
upon initiation of plasma exchange therapy, based on a diagnosis of
aTTP. Cablivi is first administered as an 11 mg intravenous
injection prior to plasma exchange, followed by an 11 mg
subcutaneous injection after completion of plasma exchange on day
1. During the daily plasma exchange period and 30 days
following daily plasma exchange, patients will take daily
11 mg subcutaneous injections. If after the initial treatment
symptoms of the underlying disease are unresolved the treatment can
be further extended for a maximum of 28 days. Subcutaneous
injection can by administered by a patient/caregiver following
proper training.
Cablivi is expected to be
available in the U.S. late in the first quarter. The U.S.
list price, or wholesale acquisition cost, for treating a typical
aTTP episode with Cablivi is $270,000. Sanofi is committed to
helping U.S. patients who have been prescribed Cablivi access their
medication and get the support they need, and will be launching
Cablivi Patient Solutions, a comprehensive patient support program.
For patients with aTTP who are prescribed Cablivi, Cablivi Patient
Solutions will provide support to eligible patients who require
financial assistance.
Cablivi was developed by Ablynx,
which was acquired by Sanofi in 2018. Cablivi was approved in
the European Union in August 2018. Cablivi is part of the
company's rare blood disorders franchise within Sanofi Genzyme, the
specialty care global business unit of Sanofi.
For full prescribing information,
please visit www.cablivi.com.
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
|
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