GSK’s drisapersen for muscular dystrophy reaches primary objective

In Clinical Development by Tim Peplow

gsk drisepersen muscular dystrophy (bunnell tm, jaeger ma, fitzsimons dp, prins kw, ervasti jm 2008)

Drisapersen, GlaxoSmithKline and Prosensa’s drug for Duchenne muscular dystrophy (DMD), achieved the primary objective in a Phase II study, according to a report from Bloomberg and an abstract on the CureDuchenne website.

“Today, GSK released the results of their Phase ll 24 and 48 week data”, writes the CureDuchenne website. “We are very pleased to report that the results are clinically significant.  It’s been a long 10 years and this drug is not yet approved, but we are looking forward to the Phase lll data later this year.”

DMD is a progressive neuromuscular disorder caused by a mutation in the dystrophin gene that leads to deficient dystrophin production. Drisapersen is an RNA-based therapy that works by “exon-skipping” – specifically it is designed to skip exon 51 in the dystrophin pre-mRNA and allow the production of dystrophin.

The abstract on the CureDuchenne website reveals that the continuous treatment arm showed a clinically meaningful and statistically significant difference from placebo on 6 Minute Walk Distance (MWD) at week 24, and at week 48, both treatment arms showed a clinically meaningful difference from placebo on 6MWD. The drug was generally well tolerated, with the majority of adverse events related to injection site reactions and proteinuria.

Drisapersen would compete with Sarepta’s eteplirsen. “Prosensa data validates Sarepta’s platform,” said Deutsche Bank analyst Robyn Karnauskas, reported by Bloomberg. Eteplirsen’s better safety profile may differentiate it from drisapersen, he said.

Read the Bloomberg report

Read the CureDuchenne abstract

Read the article on

What do you think? Do you think that these results are a positive for GSK and Prosensa, or do you think – as others have suggested – this positive data instead bodes well for Sarepta?

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