Sanofi: FDA approves Cablivi® (caplacizumab-yhdp), the first Nanobody®-based medicine, for adults with acquired thrombotic ...
06 Febrero 2019 - 10:45AM
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 |
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ir@sanofi.com |
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