Sanofi's (EPA: SAN) Cablivi (caplacizumab) has been granted marketing authorisation by the European Commission (EC) for the treatment of adults experiencing an episode of acquired thrombotic thrombocytopenic purpura (aTTP), the company disclosed on Monday.

aTTP is a rare, life-threatening, autoimmune-based blood-clotting disorder that is characterised by extensive clot formation in small blood vessels throughout the body. This results in severe thrombocytopenia (very low platelet count), microangiopathic hemolytic anaemia (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 approval makes Cablivi the first therapeutic specifically indicated for the treatment of aTTP.

The EC's decision was based on the Phase II TITAN and Phase III HERCULES studies which involved 220 adult patients with aTTP and which demonstrated the safety and efficacy of caplacizumab in addition to standard-of-care treatment, daily PEX and immunosuppression.

Cablivi was developed by Ablynx, a Sanofi company. Sanofi Genzyme, the speciality care global business unit of Sanofi, will work with the relevant local authorities to make Cablivi available to those in need in countries across Europe.

The US Food and Drug Administration (FDA) has accepted for review the Biologics License Application for caplacizumab for the treatment of patients aged 18 and over experiencing an episode of aTTP. The target action date for the decision is 6 February 2019.