Acceleron Pharma has received US Food and Drug Administration fast-track designation for ACE-031, its investigational protein therapeutic, for the treatment of Duchenne muscular dystrophy (DMD).
A double-blind, randomised, placebo-controlled Phase II trial of ACE-031 will now be carried out to evaluate its tolerability, safety and pharmacokinetics in DMD patients, who will receive simultaneous corticosteroid treatment.
The trial will also evaluate the effects of ACE-031 on muscle function, muscle mass, muscle strength, pulmonary function and quality of life.
The Phase I study of ACE-031 demonstrated a rapid increase in muscle volume and lean mass in healthy post-menopausal women.
Fast-track designation expedites the approval of new drugs which address unmet medical needs and show potential in treating life-threatening conditions.
DMD is a neuromuscular disorder, which is characterised by progressive degeneration of muscle and strength, eventually resulting in death.