Spark Ther­a­peu­tics faces a grilling as FDA pon­ders a pi­o­neer­ing OK for gene ther­a­py

With one of the most close­ly-watched FDA pan­el re­views of the year com­ing up for Spark Ther­a­peu­tics’ $ONCE lead gene ther­a­py, in­ter­nal re­view­ers at the FDA have raised some se­ri­ous ques­tions for the ex­perts to con­sid­er.

At the top of the heap of con­sid­er­a­tions the pan­el ex­perts will field on Thurs­day are ques­tions about the dura­bil­i­ty of this eye ther­a­py, the pos­si­bil­i­ty that pa­tients will need mul­ti­ple treat­ments in or­der to pre­serve vi­sion gains, the right age to use it and the use of a com­plete­ly nov­el end­point for the pri­ma­ry goal when stan­dard vi­su­al acu­ity achieve­ments fell far short of the goal on sta­tis­ti­cal sig­nif­i­cance.

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