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ARTICLE | Company News

Prosensa planning NDA for drisapersen this year

June 3, 2014 11:47 PM UTC

Prosensa Holding N.V. (NASDAQ:RNA) said it plans to submit an NDA to FDA this year seeking accelerated approval of drisapersen ( PRO051) to treat Duchenne muscular dystrophy (DMD). The compound -- an antisense oligoribonucleotide that induces exon 51 skipping on the dystrophin gene -- has breakthrough therapy designation in the U.S. for the indication. According to Prosensa, FDA said existing data could support accelerated approval.

The company said it also is committed to two confirmatory post-approval trials: a historical control trial of drisapersen and a placebo-controlled trial with a follow-on exon-skipping compound with a similar mechanism of action. On a conference call to discuss the drisapersen update, Prosensa said it plans to start the historical trial by 3Q14 and the placebo-controlled trial by as early as 1Q15. ...