Sanofi said that the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has recommended approval of its subsidiary Ablynx’s Cablivi (caplacizumab) for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP) in adults.
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.
aTTP is a life-threatening, autoimmune-based blood clotting disorder characterized by extensive clot formation in small blood vessels throughout the body, leading to thrombocytopenia, ischemia and widespread organ damage especially in the brain and heart.
There are currently no products authorised for the treatment of aTTP and despite treatment with plasma exchange (PEX) and immunosuppression, patients remain at risk for thrombotic complications, recurrences and death.
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.
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.
Source: Company Press Release