CHMP recommends approval of Cablivi for acquired thrombotic thrombocytopenic purpura.- Ablynx.

The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has recommended approval of Cablivi (caplacizumab), from Ablynx, a Sanofi Company, in Europe for the treatment of adults experiencing an episode of acquired thrombotic thrombocytopenic purpura (aTTP), a rare blood-clotting disorder. The European Commission will review the CHMP recommendation and a final decision on the Marketing Authorisation Application for Cablivi in the European Union is expected in the coming months. Directed against von Willebrand Factor (vWF), Cablivi is Ablynx's first Nanobody-based medicine to receive a positive CHMP opinion and, if approved, will be the first therapeutic specifically indicated for the treatment of aTTP.

The potential of Cablivi to address this unmet need has been demonstrated in 220 patients with aTTP, who participated in the phase II TITAN and phase III HERCULES studies. Data from the TITAN study were published in the New England Journal of Medicine in February 2016 and the positive HERCULES results were presented at the 59th Annual Meeting of the American Society of Hematology (ASH) in December 2017.

Comment: 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 Hemophilia A and B.