Drisapersen, GlaxoSmithKline's (GSK) potential exon skipping drug for boys with Duchenne muscular dystrophy has been awarded 'Breakthrough Therapy' status in the USA. The status can be awarded by the United States Food and Drug Administration - the drug regulator in the USA - to potential therapies which show encouraging results in clinical trials. In this case, the regulator used the results of a 53-patient phase II clinical trial which were reported in April. The results showed that after 24 weeks, boys with Duchenne muscular dystrophy who were given drisapersen were able to walk 35 metres further in six minutes than those given a placebo control (an inactive version of the drug).
Breakthrough status means that the company will now benefit from increased support from the FDA. This will ensure that drug development, and clinical trials can be handled as quickly and efficiently as possible.