FDA panel supports use of ports in Sarepta DMD trial

Members of FDA's Pediatric and Pediatric Ethics Advisory Committees voted 14-0 in support of allowing Sarepta Therapeutics Inc. (NASDAQ:SRPT) to use drug ports in the ongoing Phase III ESSENCE trial evaluating two therapies for Duchenne muscular dystrophy. The committee members voted unanimously that there are circumstances under which use of an indwelling central venous access device should be allowed.

Sarepta was seeking to modify the trial to use drug ports, rather than IV infusions, as the delivery mode of exon-skipping therapies SRP-4045 and SRP-4053 in patients aged seven to 13 (see BioCentury Extra, April 24)...