Sanofi has secured approval from the US Food and Drug Administration (FDA) for its blood-disorder drug Cablivi (caplacizumab-yhdp).
Cablivi has been approved in combination with plasma exchange and immunosuppression to treat acquired thrombotic thrombocytopenic purpura (aTTP) in adults. Cablivi is said to be the first FDA-approved therapy for the treatment of aTTP.
Claimed to be the first nanobody-based medicine, Cablivi targets von Willebrand factor (vWF), a protein in the blood that involves in hemostasis.
Cablivi, which is an anti-vWF Nanobody, has been designed to inhibit the interaction between vWF and platelets.
Nanobodies are a novel class of advanced therapeutic proteins based on single-domain antibody fragments, which include structural and functional properties of naturally occurring heavy chain only antibodies.
The approval of Cablivi was based on data from the Hercules multicentre, randomized, double-blind, placebo-controlled phase 3 clinical study.
Hercules study assessed the efficacy of Cablivi in combination with plasma exchange and immunosuppressive therapy against placebo, plasma exchange and immunosuppressive therapy in 145 adults with aTTP.
According to the company, the treatment with Cablivi in combination with plasma exchange and immunosuppression resulted in a significantly shorter time to platelet count response against plasma exchange and immunosuppression alone, which is a primary efficacy endpoint.
It also demonstrated a significant reduction on a composite endpoint of aTTP-related death, recurrence of aTTP and major thromboembolic event during study drug treatment against plasma exchange and immunosuppression alone.
The company intends to launch Cablivi in the US by the end of this quarter. Ablynx had developed Cablivi, which was acquired by Sanofi in 2018.
In August 2018, Cablivi secured approval in the European Union, and is part of the firm’s rare blood disorders franchise within Sanofi Genzyme.
Sanofi CEO Dr Olivier Brandicourt said: “The U.S. approval of Cablivi provides a much-needed treatment option for people facing this challenging disease. There have been limited medicines available to treat aTTP until now.
“Cablivi marks the first U.S. approval in our newly formed rare blood disorders franchise, and we look forward to continuing to provide important medicines for people living with these very serious diseases.”