BRIDGEWATER, N.J., Sept. 3, 2018 /PRNewswire/ -- (EURONEXT:
SAN) (NYSE: SNY) -- The European Commission has granted
marketing authorization for Cablivi™ (caplacizumab) for the
treatment of adults experiencing an episode of acquired thrombotic
thrombocytopenic purpura (aTTP), a rare blood-clotting disorder.
Cablivi is the first therapeutic specifically indicated for the
treatment of aTTP.
aTTP is a life-threatening, autoimmune-based blood clotting
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.
Despite the current standard-of-care treatment, consisting of
daily plasma exchange (PEX) and immunosuppression, episodes of aTTP
are still associated with a mortality rate of up to 20%, with most
deaths occurring within 30 days of
diagnosisi.
"aTTP is a devastating disease.
Many patients undergoing current standard-of-care treatment
continue to be at risk of developing acute thrombotic
complications, including stroke and heart attack, recurrence of the
disease, lack of treatment response and death," said
Marie Scully, M.D, professor of
hematology at University College London Hospitals.
"The approval of Cablivi provides an important
addition to the standard-of-care treatment for patients
with aTTP in Europe because
it can significantly reduce time to platelet count normalization
and induce a clinically meaningful reduction in
recurrences."
Cablivi was developed by Ablynx, a Sanofi company. Sanofi
Genzyme, the specialty care global business unit of Sanofi, will
work with relevant local authorities to make Cablivi available to
patients in need in countries across Europe.
Cablivi is the company's first Nanobody®-based
medicine to receive approval and the first newly approved product
that will be part of Sanofi Genzyme's Rare Blood Disorders
franchise. Earlier this year, Sanofi acquired Bioverativ which has
treatments for hemophilia A and B.
"The approval of Cablivi
provides new hope for people diagnosed with aTTP, who to date have
faced a very difficult disease with limited treatment options,"
said Bill Sibold, Executive Vice President and Head of Sanofi
Genzyme. "This approval is the next step towards
our goal of becoming the leading rare blood disorders company in
the industry. We are excited about the opportunities to continue to
expand our rare blood disorders business and to help many people
with very serious diseases."
aTTP Clinical Program and Results
The approval of Cablivi in the EU is based on the Phase II TITAN
and Phase III HERCULES studies in 220 adult patients with aTTP. The
efficacy and safety of caplacizumab in addition to standard-of-care
treatment, daily PEX and immunosuppression, were demonstrated in
these studies.
In the HERCULES study, treatment with caplacizumab in addition
to standard-of-care resulted in a significantly shorter time to
platelet count response (p<0.01), the study's primary endpoint;
a significant reduction in aTTP-related death, recurrence of aTTP,
or at least one major thromboembolic event during study drug
treatment (p<0.0001); and a significantly lower number of aTTP
recurrences in the overall study period (p<0.001). Importantly,
treatment with caplacizumab resulted in a clinically meaningful
reduction in the use of PEX and length of stay in the intensive
care unit (ICU) and the hospital, compared to the placebo
group.
In clinical trials, caplacizumab demonstrated a safety profile,
consistent with its mechanism of action. The most frequently
reported adverse reactions were epistaxis, headache and gingival
bleeding. No deaths were reported during study drug treatment in
the caplacizumab group in the TITAN and HERCULES studies, while for
the placebo group, two deaths were reported in the TITAN study and
three deaths in the HERCULES study.
U.S. FDA Review of Caplacizumab
Additionally, the U.S. Food and Drug Administration (FDA) has
accepted for priority review the Biologics License Application for
caplacizumab 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 Cablivi
Caplacizumab is a bivalent anti-vWF Nanobody that received
Orphan Drug Designation in Europe
and the United States in 2009, in
Switzerland in 2017 and in
Japan in 2018. Caplacizumab blocks
the interaction of ultralarge von Willebrand Factor (vWF) multimers
with platelets and, therefore, has an immediate effect on platelet
adhesion and the ensuing formation and accumulation of the
micro-clots that cause the severe thrombocytopenia, tissue ischemia
and organ dysfunction in aTTP.
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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i Benhamou, Y. et al., Haematologica 2012
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