Patients with the rare clotting disorder acquired thrombotic thrombocytopenic purpura (aTTP) have gained a new treatment option in Europe with the approval of Sanofi’s Cablivi.

The European Commission has authorised Cablivi (caplacizumab) as the first therapeutic specifically indicated for the treatment of adults experiencing an episode of aTTP.

The condition 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 and widespread organ damage.

Current standard-of-care is daily plasma exchange (PEX) and immunosuppression, but episodes of aTTP are still linked with a mortality rate of up to 20%, with most deaths occurring within 30 days of diagnosis, highlighting the need for new treatment options.

Data from the Phase II TITAN and Phase III HERCULES studies underpinned the drug’s approval, after showing its safety and efficacy in aTTP patients.

In the HERCULES study, treatment with caplacizumab in addition to standard-of-care significantly reduced the time to platelet count response, aTTP-related death, and recurrence of aTTP, or at least one major thromboembolic event during study drug treatment.

Also, 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,” the firm noted.

"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," said Marie Scully, professor of hematology at University College London Hospitals.

Cablivi was developed by Sanofi group Ablynx. Sanofi Genzyme will now work with relevant local authorities to make Cablivi available to patients in countries across Europe.