Sanofi's Cabrevi clears rare bleeding disorder

With Sanofi's Cablivi approval, patients with rare clotting disorders and acquired thrombocyte-reducing cyanosis have been given a new treatment option in Europe.
the European Commission has approved caplacizumab as the first treatment specifically designed to treat adult patients experiencing aTTP attack.
The condition is a life-threatening, autoimmune-based blood clotting disorder characterized by the formation of large clots in small blood vessels throughout the body, leading to severe plateplate reduction (very low plate plate count), microvascular pathogenic hemolytic anemia (loss of red blood cells through destruction), isbleed and extensive organ damage.
The current standard care is daily plasma replacement and immunosuppression, but the onset of attESTP is still associated with a mortality rate of up to 20%, and most deaths occur within 30 days of diagnosis, which emphasizes the need for new treatment options.
data from Stage 2 TITAN and Phase III HERCULES strengthened the drug's approval after showing its safety and ability in aTTP patients.
In the HERCULES study, in addition to standard treatment, the use of caplacizumab therapy significantly reduced the time for plateplate count response, and the study looked at aTTP-related deaths, aTTP recurrence, or at least one major thrombosis event during drug therapy.
in addition, the use of caplacizumab led to "less clinical significance and longer stay in intensive care units (ICU) and hospitals than in the placebo group," the company noted.
"Cablivi's approval provides an important complement to standard treatment for patients with attentives in Europe, as it significantly reduces the time it takes to normalize plate counts and induces a reduction in clinical recurrence," said Marie Scully, professor of haematology at University Hospital London.
Cablivi was developed by Sanofi Group's Ablynx. Sanofi Genzyme will work with the relevant local authorities to provide Cablivi to patients across Europe. (This net special draft)