CHMP gives positive opinion for treatment of rare blood-clotting disorder

Cablivi (caplacizumab) has received a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) for the treatment of a rare blood-clotting disorder — acquired thrombotic thrombocytopenic purpura (aTTP).

This decision will be reviewed by the European Commission, which should make its final decision on the marketing authorisation application of the treatment in the next few months.

If approved for marketing authorisation, Cablivi — developed by Ablynx, a Sanofi company — will be the first therapeutic specifically indicated for the treatment of aTTP. The condition is life-threatening and is characterised by extensive clot formation in small blood vessels throughout the body, leading to thrombocytopenia, ischaemia and widespread organ damage especially in the brain and heart.

Currently, there are no products authorised for the treatment of the autoimmune-based blood clotting disorder. The potential of Cablivi to treat the condition has been shown in 220 patients with aTTP, who took part in the Phase II TITANand Phase III HERCULESstudies.

If approved, Cablivi will be made available to patients through Sanofi Genzyme, Sanofi's specialty care business, and will be part of the unit’s new rare blood disorders franchise that will launch in 2019 and which will also include Bioverativ's treatments for Haemophilia A and B.