FDA lifts clin­i­cal hold on Sarep­ta's Duchenne MD gene ther­a­py, clear­ing the way to a quick launch of a piv­otal tri­al

In a swift turn­around, Sarep­ta an­nounced that it has cleared the FDA’s con­cerns about its gene ther­a­py pro­gram for Duchenne mus­cu­lar dy­s­tro­phy, con­vinc­ing reg­u­la­tors to lift a clin­i­cal hold is­sued just two months ago.

To­geth­er with col­lab­o­ra­tors at the Na­tion­wide Chil­dren’s Hos­pi­tal, the com­pa­ny sub­mit­ted an ac­tion plan to ad­dress the “pres­ence of trace amounts of DNA frag­ment in re­search-grade third-par­ty sup­plied plas­mid in a man­u­fac­tur­ing lot” — which they say trig­gered the hold. The part­ners of­fered up “an au­dit of the plas­mid sup­pli­er and a com­mit­ment to use GMP-s plas­mid for all fu­ture pro­duc­tion lots.”

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