Positive results for Cablivi in rare blood clotting disorder

Positive results of the Phase III trial of Cablivi (caplacizumab) in adults with acquired thrombotic thrombocytopenic purpura (aTTP) have been published in the New England Journal of Medicine (NEJM).

Under development at French pharma major Sanofi (Euronext: SAN), Cablivi gained European approval in August last year and is under review in the USA, with a Food and Drug Administration decision expected February 6, 2019. Cablivi is the first therapeutic specifically indicated for the treatment of 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.