Cablivi approved for acquired thrombotic thrombocytopenic purpura (aTTP)

The European Commission has granted marketing authorisation for Cablivi (caplacizumab) 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 characterised 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.

Despite 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.

Cablivi was developed by Ablynx, a Sanofi company. Sanofi Genzyme, the specialty care global business unit of Sanofi, will work with relevant local authorities to make Cablivi available to patients in need in countries across Europe. Cablivi is the company’s first Nanobody®-based medicine to receive approval.

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. In the HERCULES study, treatment with caplacizumab in addition to standard-of-care resulted in a significantly shorter time to platelet count response, a significant reduction in aTTP-related death, recurrence of aTTP, or at least one major thromboembolic event during study drug treatment and a significantly lower number of aTTP recurrences in the overall study period.

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.

Marie Scully, M.D, professor of hematology at University College London Hospitals said, “aTTP is a devastating disease. Many patients undergoing current standard-of-care treatment continue to be at risk of developing acute thrombotic complications, including stroke and heart attack, recurrence of the disease, lack of treatment response and death. The approval of Cablivi provides an important addition to the standard-of-care treatment for patients with aTTP in Europe because it can significantly reduce time to platelet count normalization and induce a clinically meaningful reduction in recurrences.”