New England Journal of Medicine publishes positive results of the pivotal trial of Cablivi® (caplacizumab) for rare blood clotting disorder
January 9, 2019Download PDF
| New England Journal of Medicine publishes positive results of the pivotal trial of Cablivi® (caplacizumab) for rare blood clotting disorder|
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:
"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.
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