The US Food and Drug Administration (FDA) has granted breakthrough therapy designation to GlaxoSmithKline’s drisapersen for the potential treatment of patients with Duchenne muscular dystrophy. The agency has concluded that the drug qualifies as breakthrough based on a Phase II study of drisapersen, which is licensed from Prosensa of the Netherlands. GSK is looking at drisapersen in ambulant (Phases II and III) and non-ambulant boys (Phase I) with DMD who have dystrophin gene mutations which are amenable to an exon 51 skip (up to 13% of all patients). Breakthrough therapy designation is distinct from the FDA’s other fast-track programmes, such as accelerated approval and priority review, as it involves more intensive guidance from the agency on putting together an efficient drug development programme.
