BioMarin Pharmaceuticals has filed experimental Duchenne muscular dystrophy drug drisapersen for approval in Europe, raising hopes for the first approved treatment for the condition.
Drisapersen is an investigational antisense oligonucleotide drug being developed for the treatment of the largest subset of DMD amenable to single exon skipping, which BioMarin acquired via its $840-million purchase of Prosensa in November last year.
The drug, which also had a stint at GlaxoSmithKline until a failed Phase III trial spurred the drug giant to hand back rights to Prosensa, has also been filed via a rolling New Drug Application in the US.
In the trial, drisapersen failed to meet the primary endpoint in the GSK study of a statistically significant improvement of distance walked in six minutes compared to placebo.
But subsequent analyses of aggregate data from the entire clinical programme showed a 49-metre difference between those on continual treatment (n=52) and those who had been on placebo for 48 weeks followed by active drug (n=31), re-igniting confidence in the drug.
DMD is the most common fatal genetic disorder diagnosed in childhood, affecting around one in every 3,500 live male births with about 20,000 new cases diagnosed globally each year.