Sarepta planning NDA for DMD candidate casimersen

Sarepta said it plans to submit an NDA to FDA in mid-2019 for casimersen (SRP-4045) to treat Duchenne muscular dystrophy amenable to exon 45 skipping. The company will seek approval for casimersen based on a surrogate endpoint of an increase in dystrophin protein that was reported in an interim analysis of the Phase III ESSENCE trial.

Among 27 evaluable DMD patients in ESSENCE, Sarepta Therapeutics Inc. (NASDAQ:SRPT) said once-weekly 30 mg/kg IV casimersen Increased mean dystrophin protein levels to 1.736% of normal at week 48 compared with 0.925% of normal at baseline as measured by Western blot (p<0.001)...