LONDON, June 27 GlaxoSmithKline said on
Thursday U.S. regulators have given breakthrough therapy status
for its investigational compound drisapersen for the potential
treatment of patients with duchenne muscular dystrophy (DMD).
The U.S. Food and Drug Administration (FDA) created the
"breakthrough therapy" designation earlier this year for
medicines deemed likely to demonstrate "substantial improvement"
over existing drugs.
DMD is a debilitating childhood neuromuscular disease that
affects 1 in 3,500 live male births, GSK said, adding that the
disease was caused by mutations in the dystrophin gene.
GSK said its clinical development plan evaluates the effect
of drisapersen in ambulant (Phases II and III) and non-ambulant
boys (Phase I) with DMD who have certain dystrophin gene