A first-of-its-kind organization launched on May 13 unifying the voices of life science companies committed to delivering desperately needed treatments to rare disease patients. The Rare Disease Company Coalition’s 10 founding members have brought 22 treatments to market and have more than 160 rare disease programs in the works. 

Cambridge, Massachusetts-based Acceleron Pharma announced that the company’s Phase II clinical trial of ACE-083 in patients with Charcot-Marie-Tooth disease (CMT) failed to show functional improvement.

The U.S. FDA approved Celgene and Acceleron Pharma’s Reblozyl for the treatment of anemia in adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions.

Celgene and developmental partner Acceleron Pharma are eying a potential U.S. FDA approval of a blood-disease treatment.