Sarepta seems to have the edge in race for Duchenne drug

In Clinical Development by Tim Peplow

duchenne muscular dystrophy sarepta gsk (kuebi)

There’s a race on, and the pace has just been upped. Which drug will be first to be approved for Duchenne muscular dystrophy (DMD)? Will it be Sarepta’s eteplirsen or GSK/Prosensa’s drisapersen? Both are exon 51 skipping drugs, and both getting Wall Street talking. The latest surge in interest surrounds the release of data from GSK/Prosensa’s mid-stage trials. While the data showed that the GSK/Prosensa drug produced dystrophin in 72% of boys who received a continuous dose and 59% of boys who received an intermittent dose, it was Sarepta’s shares that bounced up.

While the drisapersen results are enormously encouraging for the 300,000 boys and young men with DMD worldwide, the GSK/Prosensa results are viewed as “disappointing” when compared to Sarepta’s 100% figures – albeit in a much smaller phase II trial.

Analysts have pointed out that the drisapersen data shuffles Sarepta into the lead. “We believe this introduces new clinical, regulatory and commercial risk for this competitive drug, which should benefit Sarepta given the more favorable therapeutic index that eteplirsen appears to have,” said Baird analyst Brian Skorney.

Sarepta and GSK are not the only companies developing orphan drugs for DMD however – click here to read about the 12 organisations with an approved designation for an orphan medicinal product to treat DMD.

Read more at Reuters >

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