Drisapersen given breakthrough therapy status

Muscular Dystrophy Campaign
61A Great Suffolk Street
London
SE1 0BU 

Monday 1st July 2013.

Drisapersen given breakthrough therapy status          

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 (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.

Robert Meadowcroft, Chief Executive of the Muscular Dystrophy Campaign, said:  

"This is very encouraging news and we are one more step closer to delivering a potential treatment to some boys living with Duchenne muscular dystrophy. "

"We are also working closely with the All Party Parliamentary Group on muscular dystrophy who are conducting an Inquiry into access to potential treatments for conditions such as Duchenne muscular dystrophy. The Inquiry will look at the "Breakthrough Therapy" designation from the FDA and consider whether similar approaches could be used by regulators here in the UK."