European Commission approves Cablivi to treat acquired thrombotic thrombocytopenic purpura (aTTP)- Sanofi

The European Commission has granted marketing authorization for Cablivi (caplacizumab) from Sanofi 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. espite 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 diagnosis.

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 studys 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.