FDA's CBER chief hints at ex­pand­ing Sarep­ta's gene ther­a­py la­bel, sends stock up 25%

De­spite a failed con­fir­ma­to­ry tri­al an­nounced in Oc­to­ber, the FDA’s Pe­ter Marks, the top reg­u­la­tor for all cell and gene ther­a­pies in the US, is sig­nal­ing a like­ly ex­pand­ed la­bel for Sarep­ta’s Duchenne gene ther­a­py, send­ing the stock up by about 25% over the past five days.

While it’s rare to see a reg­u­la­tor be so sup­port­ive of a sin­gle com­pa­ny, it’s rar­er still to see Marks sig­nal­ing a like­ly ex­pand­ed ap­proval for Sarep­ta’s Ele­v­idys, which is cur­rent­ly on­ly au­tho­rized un­der the ac­cel­er­at­ed path­way for am­bu­la­to­ry pe­di­atric pa­tients aged 4 through 5 years with Duchenne, but which the com­pa­ny would like to ex­tend to all Duchenne pa­tients with­out re­stric­tion to age or am­bu­la­to­ry sta­tus.

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