Posted by Michael Wonder on 06 Feb 2019
FDA approves first therapy for the treatment of adult patients with a rare blood clotting disorder
6 February 2019 - The U.S. FDA today approved Cablivi (caplacizumab-yhdp) injection, the first therapy specifically indicated, in combination with plasma exchange and immunosuppressive therapy, for the treatment of adult patients with acquired thrombotic thrombocytopenic purpura (aTTP), a rare and life-threatening disorder that causes blood clotting.
The efficacy of Cablivi was studied in a clinical trial of 145 patients who were randomized to receive either Cablivi or a placebo.
The FDA granted this application priority review designation. Cablivi also received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.
