FDA approves Cablivi (caplacizumab-yhdp), the first Nanobody-based medicine, for adults with acquired thrombotic thrombocytopenic purpura (aTTP)

PARIS  – 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 naturallyoccurring 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
Investor Relations Contact
George Grofik
Tel.: +33 (0)1 53 77 45 45
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ut 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

Vartika Singh

Vartika Singh is a content writer who loves to write research articles and reports at PharmaShots. She has in-depth knowledge of the life sciences industry including the Pharma and Biotech sectors. Any articles written by her can be contacted at connect@pharmashots.com.

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