Cablivi is claimed to be the first therapeutic specifically developed to treat aTTP, a rare blood-clotting disorder.
The approval was based on data from the phase II Titan and Phase III Hercules studies in 220 adult patients with aTTP.
According to Sanofi, caplacizumab showed a safety profile and consistent with its mechanism of action in clinical studies.
Sanofi company Ablynx has developed Cablivi, while other unit Sanofi Genzyme will coordinate with relevant local authorities to provide Cablivi to the required patients across Europe.
Cablivi, which is the firm’s first Nanobody-based medicine to secure approval, will be part of Sanofi Genzyme’s rare blood disorders franchise.
Caplacizumab is a bivalent anti-vWF Nanobody, which secured orphan drug designation in Europe and the US in 2009, while in Switzerland and Japan in 2017 and 2018.
Caplacizumab inhibits the interaction of ultralarge von Willebrand Factor (vWF) multimers with platelets, enabling to have an immediate effect on platelet adhesion.
aTTP is a autoimmune-based blood clotting disorder indicated by extensive clot formation in small blood vessels across the body.
It may result in severe thrombocytopenia (very low platelet count), microangiopathic hemolytic anemia (loss of red blood cells through destruction), ischemia (restricted blood supply to parts of the body) and widespread organ damage specifically in the brain and heart.
At the starting of this year, Sanofi acquired Bioverativ that provides treatments for hemophilia A and B.
Sanofi Genzyme head and executive vice president Bill Sibold said: “The approval of Cablivi provides new hope for people diagnosed with aTTP, who to date have faced a very difficult disease with limited treatment options.
“This approval is the next step towards our goal of becoming the leading rare blood disorders company in the industry. We are excited about the opportunities to continue to expand our rare blood disorders business and to help many people with very serious diseases.”