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.
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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.
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