Posted by Michael Wonder on 09 Nov 2019
FDA approves first therapy to treat patients with rare blood disorder
8 November 2019 - Today the U.S. FDA granted approval to Reblozyl (luspatercept–aamt) for the treatment of anaemia (lack of red blood cells) in adult patients with beta thalassaemia who require regular red blood cell (RBC) transfusions.
The approval of Reblozyl was based on the results of a clinical trial of 336 patients with beta thalassemia who required RBC transfusions, of which 112 received a placebo.
The FDA granted this application fast track designation. Reblozyl also received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. The FDA granted approval of Reblozyl to Celegene Corporation.
