FDA fast tracks Acceleron muscle disease drug target
Acceleron Pharma Inc. has announced that it has received fast track designation from the U.S. Food and Drug Administration for a drug candidate designed to build muscle mass and strength. The therapeutic, ACE-031, was developed to treat Duchenne Muscular Dystrophy (DMD), a fatal neuromuscular disease marked by patient’s loss of muscle mass and strength over time.
The fast track designation makes the drug candidate eligible for priority review by the FDA. ACE-031 is an investigational protein therapeutic that has shown promise in increasing muscle mass, strength and physical use in animal models with muscle disease.
Acceleron is conducting a Phase 2 trial of ACE-031, in conjunction with corticosteroid treatment, in DMD patients. The trial, taking place in Canada, is expected to test the safety, tolerability and pharmacokinetics of the drug candidate.
The privately held Cambridge biotech was co-founded in 2004 by former Mass High Tech All-Star Christoph Westphal, head of GlaxoSmithKline PLC’s Centre of Excellence for External Drug Discovery. In June, Acceleron raised $8.4 million of a planned $11.5 million securities offering, according to a regulatory filing with the U.S. Securities and Exchange Commission.