The New England Journal of Medicines published positive results of Phase 3 trial of Cablivi medicine in adults for a rare blood clotting disorder on 9 January 2019.
A clinical trial of 145 patients started in November 2015. The course of the study consisted of three phases. Phase 3, named HERCULES reached its endpoint with results showing a significant increase in blood count platelets when treated with Cablivi.
The results of the Phase 3 trial of Cablivi demonstrated that treatment with Cablivi helped reduce the time to platelet count normalization in patients suffering from a rare blood clotting disorder, acquired thrombotic thrombocytopenic purpura (aTTP).
aTTP is a critical and rare autoimmune blood disorder which involves the extensive formation of blood clots in blood vessels. aTTP leads to very low platelet count, loss of red blood cells, restricted blood supply to parts of the body and organ damage especially affecting the brain and heart.
Treatment with Cablivi resulted in a 74% reduction in aTTP-related death and 67% reduction in the recurrence of aTTP. Also, normalization of several organ-damage markers is reported to occur sooner in patients receiving Cablivi.
The already existing treatment consists of daily plasma exchange, in which the patient's plasma is removed and replaced with donor's plasma. With such treatments, patients continue to be at risk for acute blood clotting resulting in heart attacks and stroke.
With Cablivi treatment, the use of plasma exchange in patients is reduced significantly, with a shorter medical stay in the hospitals and ICUs.
Cablivi is a medicine that prevents clotting of blood vessels resulting in proper blood flow in the body. It is the first medicinal treatment for adults used for treating aTTP.
"aTTP is a life-threatening disease. 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, the lead author of the HERCULES study, M.D, and professor of hematology at University College London Hospitals
Professor Scully also said, "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."
“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. Sanofi Genzyme is a global business unit of Sanofi.
Cablivi was approved by the European Commission in August 2018 for the treatment of adult patients suffering from aTTP.
Cablivi is developed by Ablynx, a Sanofi company. Sanofi is a biopharmaceutical company that focuses on human health. Sanofi provides vaccines and treatments for illnesses and aims to help people fight from rare diseases.
With Cablivi treatment being available, it is a step forward in the field of medicine and a step towards helping patients suffering from a severe blood clotting disorder.