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Eteplirsen Revisited? FDA Panel To Weigh Protocol Changes For Two Sarepta Drugs

 
• By Sue Sutter

US agency’s advisory committees will consider whether ongoing trial of exon 45- and 53-skipping compounds for Duchenne muscular dystrophy should allow use of in-dwelling ports to aid infusion.

FDA entrance sign 2016

Eight months after the controversial approval of Sarepta Therapeutics Inc.’s Exondys 51 (eteplirsen), the US FDA is convening its external advisors to weigh changes in an ongoing trial involving two of the company’s investigational Duchenne muscular dystrophy (DMD) treatments.

On May 18, FDA’s Pediatric Advisory Committee and Pediatric Ethics Subcommittee will meet to consider whether in-dwelling drug ports should be allowed for DMD patients in Sarepta’s ESSENCE trial. FDA...

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